메뉴 건너뛰기




Volumn 11, Issue 4, 2014, Pages 817-839

Gene Therapy for the Nervous System: Challenges and New Strategies

Author keywords

Central nervous system; Gene delivery; Gene therapy; Nonviral gene delivery; Virus vector

Indexed keywords

ABC TRANSPORTER; AROMATIC LEVO AMINO ACID DECARBOXYLASE; CEREBROSIDE SULFATASE; CYTOSINE DEAMINASE; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; N SULPHOGLUCOSAMINE SULPHOHYDROLASE; NERVE GROWTH FACTOR; SULFATASE; SULFATASE MODIFYING FACTOR 1; THYMIDINE KINASE; TRANSCRIPTOME; UNCLASSIFIED DRUG;

EID: 84919778470     PISSN: 19337213     EISSN: 18787479     Source Type: Journal    
DOI: 10.1007/s13311-014-0299-5     Document Type: Review
Times cited : (74)

References (257)
  • 1
    • 84877578223 scopus 로고    scopus 로고
    • Progress in gene therapy for neurological disorders
    • PID: 23609618, COI: 1:CAS:528:DC%2BC3sXnsVWitLw%3D
    • Simonato M, Bennett J, Boulis NM, et al. Progress in gene therapy for neurological disorders. Nat Rev Neurol 2013;9:277–291.
    • (2013) Nat Rev Neurol , vol.9 , pp. 277-291
    • Simonato, M.1    Bennett, J.2    Boulis, N.M.3
  • 2
    • 84886290228 scopus 로고    scopus 로고
    • Recent gene therapy advancements for neurological diseases
    • PID: 23449113
    • Nagabhushan Kalburgi S, Khan NN, Gray SJ. Recent gene therapy advancements for neurological diseases. Discov Med 2013;15:111–119.
    • (2013) Discov Med , vol.15 , pp. 111-119
    • Nagabhushan Kalburgi, S.1    Khan, N.N.2    Gray, S.J.3
  • 3
    • 0033585514 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression
    • PID: 10022545, COI: 1:CAS:528:DyaK1MXhtVKnur4%3D
    • Lo WD, Qu G, Sferra TJ, et al. Adeno-associated virus-mediated gene transfer to the brain: duration and modulation of expression. Hum Gene Ther 1999;10:201–213.
    • (1999) Hum Gene Ther , vol.10 , pp. 201-213
    • Lo, W.D.1    Qu, G.2    Sferra, T.J.3
  • 5
    • 33746883603 scopus 로고    scopus 로고
    • Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging
    • PID: 16820324, COI: 1:CAS:528:DC%2BD28XotFyksrg%3D
    • Deroose CM, Reumers V, Gijsbers R, et al. Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging. Mol Ther 2006;14:423–431.
    • (2006) Mol Ther , vol.14 , pp. 423-431
    • Deroose, C.M.1    Reumers, V.2    Gijsbers, R.3
  • 6
    • 77649096721 scopus 로고    scopus 로고
    • A limited innate immune response is induced by a replication-defective herpes simplex virus vector following delivery to the murine central nervous system
    • PID: 20095947, COI: 1:CAS:528:DC%2BC3cXlt12nu70%3D
    • Zeier Z, Aguilar JS, Lopez CM et al. A limited innate immune response is induced by a replication-defective herpes simplex virus vector following delivery to the murine central nervous system. J Neurovirol 2009;15:411–424.
    • (2009) J Neurovirol , vol.15 , pp. 411-424
    • Zeier, Z.1    Aguilar, J.S.2    Lopez, C.M.3
  • 7
    • 0034331161 scopus 로고    scopus 로고
    • Pretreatment with protease is a useful experimental strategy for enhancing adenovirus-mediated cancer gene therapy
    • PID: 11084679, COI: 1:CAS:528:DC%2BD3cXnvVOgsbs%3D
    • Kuriyama N, Kuriyama H, Julin CM, Lamborn K, Israel MA. Pretreatment with protease is a useful experimental strategy for enhancing adenovirus-mediated cancer gene therapy. Hum Gene Ther 2000;11:2219–2230.
    • (2000) Hum Gene Ther , vol.11 , pp. 2219-2230
    • Kuriyama, N.1    Kuriyama, H.2    Julin, C.M.3    Lamborn, K.4    Israel, M.A.5
  • 8
    • 0031666608 scopus 로고    scopus 로고
    • Pre-treatment with elastase improves the efficiency of percutaneous adenovirus-mediated gene transfer to the arterial media
    • PID: 10326024, COI: 1:CAS:528:DyaK1cXltFOjtr4%3D
    • Maillard L, Ziol M, Tahlil O, et al. Pre-treatment with elastase improves the efficiency of percutaneous adenovirus-mediated gene transfer to the arterial media. Gene Ther 1998;5:1023–1030.
    • (1998) Gene Ther , vol.5 , pp. 1023-1030
    • Maillard, L.1    Ziol, M.2    Tahlil, O.3
  • 9
    • 0033847140 scopus 로고    scopus 로고
    • Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle
    • PID: 10981669, COI: 1:CAS:528:DC%2BD3cXlvFajs7c%3D
    • Favre D, Cherel Y, Provost N, et al. Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle. Gene Ther 2000;7:1417–1420.
    • (2000) Gene Ther , vol.7 , pp. 1417-1420
    • Favre, D.1    Cherel, Y.2    Provost, N.3
  • 10
    • 2442572127 scopus 로고    scopus 로고
    • Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain
    • PID: 15144577, COI: 1:CAS:528:DC%2BD2cXjvF2ls7k%3D
    • Hadaczek P, Mirek H, Bringas J, Cunningham J, Bankiewicz K. Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain. Hum Gene Ther 2004;15:469–479.
    • (2004) Hum Gene Ther , vol.15 , pp. 469-479
    • Hadaczek, P.1    Mirek, H.2    Bringas, J.3    Cunningham, J.4    Bankiewicz, K.5
  • 11
    • 34247199357 scopus 로고    scopus 로고
    • Imaging the modulation of adenoviral kinetics and biodistribution for cancer gene therapy
    • PID: 17356543, COI: 1:CAS:528:DC%2BD2sXhtVejt7bP
    • Mocanu JD, Yip KW, Alajez NM, et al. Imaging the modulation of adenoviral kinetics and biodistribution for cancer gene therapy. Mol Ther 2007;15:921–929.
    • (2007) Mol Ther , vol.15 , pp. 921-929
    • Mocanu, J.D.1    Yip, K.W.2    Alajez, N.M.3
  • 12
    • 84855906863 scopus 로고    scopus 로고
    • Distribution properties of lentiviral vectors administered into the striatum by convection-enhanced delivery
    • PID: 21793715, COI: 1:CAS:528:DC%2BC38XoslSrsg%3D%3D
    • White E, Bienemann A, Megraw L, et al. Distribution properties of lentiviral vectors administered into the striatum by convection-enhanced delivery. Hum Gene Ther 2012;23:115–127.
    • (2012) Hum Gene Ther , vol.23 , pp. 115-127
    • White, E.1    Bienemann, A.2    Megraw, L.3
  • 13
    • 33645452310 scopus 로고    scopus 로고
    • Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain
    • PID: 16544978, COI: 1:CAS:528:DC%2BD28XisFWgu7k%3D
    • Hadaczek P, Kohutnicka M, Krauze MT, et al. Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum Gene Ther 2006;17:291–302.
    • (2006) Hum Gene Ther , vol.17 , pp. 291-302
    • Hadaczek, P.1    Kohutnicka, M.2    Krauze, M.T.3
  • 14
    • 60549099179 scopus 로고    scopus 로고
    • Efficient gene therapy-based method for the delivery of therapeutics to primate cortex
    • PID: 19193857, COI: 1:CAS:528:DC%2BD1MXit12gu74%3D
    • Kells AP, Hadaczek P, Yin D, et al. Efficient gene therapy-based method for the delivery of therapeutics to primate cortex. Proc Natl Acad Sci USA 2009;106:2407–2411.
    • (2009) Proc Natl Acad Sci USA , vol.106 , pp. 2407-2411
    • Kells, A.P.1    Hadaczek, P.2    Yin, D.3
  • 15
    • 34548610909 scopus 로고    scopus 로고
    • A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease
    • PID: 17855607, COI: 1:CAS:528:DC%2BD2sXhtVyksbjI
    • Cearley CN, Wolfe JH. A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J Neurosci 2007;27:9928–9940.
    • (2007) J Neurosci , vol.27 , pp. 9928-9940
    • Cearley, C.N.1    Wolfe, J.H.2
  • 16
    • 34247271439 scopus 로고    scopus 로고
    • Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain
    • PID: 17343566, COI: 1:CAS:528:DC%2BD2sXjvVKjtLg%3D
    • Taymans JM, Vandenberghe LH, Haute CV, et al. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther 2007;18:195–206.
    • (2007) Hum Gene Ther , vol.18 , pp. 195-206
    • Taymans, J.M.1    Vandenberghe, L.H.2    Haute, C.V.3
  • 17
    • 67651091377 scopus 로고    scopus 로고
    • Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-beta
    • PID: 19197327, COI: 1:CAS:528:DC%2BD1MXhsFeqtrw%3D
    • Meijer DH, Maguire CA, LeRoy SG, Sena-Esteves M. Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-beta. Cancer Gene Ther 2009;16:664–671.
    • (2009) Cancer Gene Ther , vol.16 , pp. 664-671
    • Meijer, D.H.1    Maguire, C.A.2    LeRoy, S.G.3    Sena-Esteves, M.4
  • 18
    • 84877847142 scopus 로고    scopus 로고
    • Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates
    • PID: 23517473, COI: 1:CAS:528:DC%2BC3sXns1ahs74%3D
    • Samaranch L, Salegio EA, San Sebastian W, et al. Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates. Hum Gene Ther 2013;24:526–532.
    • (2013) Hum Gene Ther , vol.24 , pp. 526-532
    • Samaranch, L.1    Salegio, E.A.2    San Sebastian, W.3
  • 19
    • 41149092265 scopus 로고    scopus 로고
    • Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis
    • PID: 18362923, COI: 1:CAS:528:DC%2BD1cXjslCnt78%3D
    • Chang M, Cooper JD, Sleat DE, et al. Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis. Mol Ther 2008;16:649–656.
    • (2008) Mol Ther , vol.16 , pp. 649-656
    • Chang, M.1    Cooper, J.D.2    Sleat, D.E.3
  • 20
    • 80053574021 scopus 로고    scopus 로고
    • Treatment of brain inflammatory diseases by delivering exosome encapsulated anti-inflammatory drugs from the nasal region to the brain
    • PID: 21915101, COI: 1:CAS:528:DC%2BC3MXhtFGnurjL
    • Zhuang X, Xiang X, Grizzle W, et al. Treatment of brain inflammatory diseases by delivering exosome encapsulated anti-inflammatory drugs from the nasal region to the brain. Mol Ther 2011;19:1769–1779.
    • (2011) Mol Ther , vol.19 , pp. 1769-1779
    • Zhuang, X.1    Xiang, X.2    Grizzle, W.3
  • 21
    • 60149106907 scopus 로고    scopus 로고
    • Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
    • PID: 19098898, COI: 1:CAS:528:DC%2BD1cXhsFajtbbJ
    • Foust KD, Nurre E, Montgomery CL, et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2009;27:59–65.
    • (2009) Nat Biotechnol , vol.27 , pp. 59-65
    • Foust, K.D.1    Nurre, E.2    Montgomery, C.L.3
  • 22
    • 12344273726 scopus 로고    scopus 로고
    • The blood-brain barrier: bottleneck in brain drug development
    • PID: 15717053
    • Pardridge WM. The blood-brain barrier: bottleneck in brain drug development. NeuroRx 2005;2:3–14.
    • (2005) NeuroRx , vol.2 , pp. 3-14
    • Pardridge, W.M.1
  • 23
    • 0034782616 scopus 로고    scopus 로고
    • Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors
    • PID: 11593358, COI: 1:CAS:528:DC%2BD3MXnt1eksbw%3D
    • Peng KW, Pham L, Ye H, et al. Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors. Gene Ther 2001;8:1456–1463.
    • (2001) Gene Ther , vol.8 , pp. 1456-1463
    • Peng, K.W.1    Pham, L.2    Ye, H.3
  • 24
    • 0034980914 scopus 로고    scopus 로고
    • Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver
    • PID: 11162308, COI: 1:CAS:528:DC%2BD38XhvFKjtLY%3D
    • Tao N, Gao GP, Parr M, et al. Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver. Mol Ther 2001;3:28–35.
    • (2001) Mol Ther , vol.3 , pp. 28-35
    • Tao, N.1    Gao, G.P.2    Parr, M.3
  • 25
    • 44349170706 scopus 로고    scopus 로고
    • Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection
    • PID: 18414476, COI: 1:CAS:528:DC%2BD1cXmt1OrsrY%3D
    • Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. Analysis of AAV serotypes 1–9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008;16:1073–1080.
    • (2008) Mol Ther , vol.16 , pp. 1073-1080
    • Zincarelli, C.1    Soltys, S.2    Rengo, G.3    Rabinowitz, J.E.4
  • 26
    • 80053444500 scopus 로고    scopus 로고
    • Rapid and efficient clearance of blood-borne virus by liver sinusoidal endothelium
    • PID: 21980295, COI: 1:CAS:528:DC%2BC3MXhtlCmtr7O
    • Ganesan LP, Mohanty S, Kim J, et al. Rapid and efficient clearance of blood-borne virus by liver sinusoidal endothelium. PLoS Pathog 2011;7:e1002281.
    • (2011) PLoS Pathog , vol.7 , pp. e1002281
    • Ganesan, L.P.1    Mohanty, S.2    Kim, J.3
  • 27
    • 12744278445 scopus 로고    scopus 로고
    • Differences in transductional tropism of adenoviral and lentiviral vectors in the rat brainstem
    • PID: 15542614, COI: 1:CAS:528:DC%2BD2MXht1ymtL4%3D
    • Duale H, Kasparov S, Paton JF, Teschemacher AG. Differences in transductional tropism of adenoviral and lentiviral vectors in the rat brainstem. Exp Physiol 2005;90:71–78.
    • (2005) Exp Physiol , vol.90 , pp. 71-78
    • Duale, H.1    Kasparov, S.2    Paton, J.F.3    Teschemacher, A.G.4
  • 28
    • 33846015516 scopus 로고    scopus 로고
    • Transduction of brain by herpes simplex virus vectors
    • PID: 17164771, COI: 1:CAS:528:DC%2BD2sXhtVejurnO
    • Berges BK, Wolfe JH, Fraser MW. Transduction of brain by herpes simplex virus vectors. Mol Ther 2007;15:20–29.
    • (2007) Mol Ther , vol.15 , pp. 20-29
    • Berges, B.K.1    Wolfe, J.H.2    Fraser, M.W.3
  • 29
    • 84878568088 scopus 로고    scopus 로고
    • SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors
    • PID: 23358188, COI: 1:CAS:528:DC%2BC3sXhsFCgu70%3D
    • Piccolo P, Vetrini F, Mithbaokar P, et al. SR-A and SREC-I are Kupffer and endothelial cell receptors for helper-dependent adenoviral vectors. Mol Ther 2013;21:767–774.
    • (2013) Mol Ther , vol.21 , pp. 767-774
    • Piccolo, P.1    Vetrini, F.2    Mithbaokar, P.3
  • 30
    • 67651006589 scopus 로고    scopus 로고
    • Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
    • PID: 19436115, COI: 1:CAS:528:DC%2BD1MXntVCjtLc%3D
    • Pien GC, Basner-Tschakarjan E, Hui DJ, et al. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest 2009;119:1688–95.
    • (2009) J Clin Invest , vol.119 , pp. 1688-1695
    • Pien, G.C.1    Basner-Tschakarjan, E.2    Hui, D.J.3
  • 31
    • 58849128187 scopus 로고    scopus 로고
    • Cytotoxic T lymphocyte responses to transgene product, not adeno-associated viral capsid protein, limit transgene expression in mice
    • PID: 18828728, COI: 1:CAS:528:DC%2BD1MXhtVehtbg%3D
    • Siders WM, Shields J, Kaplan J, et al. Cytotoxic T lymphocyte responses to transgene product, not adeno-associated viral capsid protein, limit transgene expression in mice. Hum Gene Ther 2009;20:11–20.
    • (2009) Hum Gene Ther , vol.20 , pp. 11-20
    • Siders, W.M.1    Shields, J.2    Kaplan, J.3
  • 32
    • 66149123049 scopus 로고    scopus 로고
    • Adenovirus activates complement by distinctly different mechanisms in vitro and in vivo: indirect complement activation by virions in vivo
    • PID: 19321608, COI: 1:CAS:528:DC%2BD1MXmtlGks7o%3D
    • Tian J, Xu Z, Smith JS, et al. Adenovirus activates complement by distinctly different mechanisms in vitro and in vivo: indirect complement activation by virions in vivo. J Virol 2009;83:5648–5658.
    • (2009) J Virol , vol.83 , pp. 5648-5658
    • Tian, J.1    Xu, Z.2    Smith, J.S.3
  • 33
    • 33344466244 scopus 로고    scopus 로고
    • Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice
    • PID: 16249376, COI: 1:CAS:528:DC%2BD28XitFChsb4%3D
    • Scallan CD, Jiang H, Liu T, et al. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood 2006;107:1810–1817.
    • (2006) Blood , vol.107 , pp. 1810-1817
    • Scallan, C.D.1    Jiang, H.2    Liu, T.3
  • 34
    • 84895546715 scopus 로고    scopus 로고
    • Pre-clinical assessment of immune responses to adeno-associated virus (AAV) vectors
    • PID: 24570676
    • Basner-Tschakarjan E, Bijjiga E, Martino AT. Pre-clinical assessment of immune responses to adeno-associated virus (AAV) vectors. Front Immunol 2014;5:28.
    • (2014) Front Immunol , vol.5 , pp. 28
    • Basner-Tschakarjan, E.1    Bijjiga, E.2    Martino, A.T.3
  • 35
    • 84867070624 scopus 로고    scopus 로고
    • Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies
    • PID: 22735381, COI: 1:CAS:528:DC%2BC38Xpt1ShsLY%3D
    • Treleaven CM, Tamsett TJ, Bu J, et al. Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies. Mol Ther 2012;20:1713–1723.
    • (2012) Mol Ther , vol.20 , pp. 1713-1723
    • Treleaven, C.M.1    Tamsett, T.J.2    Bu, J.3
  • 36
    • 84875925195 scopus 로고    scopus 로고
    • Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
    • PID: 23303281, COI: 1:CAS:528:DC%2BC3sXls1OqsQ%3D%3D
    • Gray SJ, Nagabhushan Kalburgi S, McCown TJ, Jude Samulski R. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther 2013;20:450–459.
    • (2013) Gene Ther , vol.20 , pp. 450-459
    • Gray, S.J.1    Nagabhushan Kalburgi, S.2    McCown, T.J.3    Jude Samulski, R.4
  • 37
    • 0034986065 scopus 로고    scopus 로고
    • Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain
    • PID: 11162309, COI: 1:CAS:528:DC%2BD38XhvFKjtLc%3D
    • Thomas CE, Birkett D, Anozie I, Castro MG, Lowenstein PR. Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol Ther 2001;3:36–46.
    • (2001) Mol Ther , vol.3 , pp. 36-46
    • Thomas, C.E.1    Birkett, D.2    Anozie, I.3    Castro, M.G.4    Lowenstein, P.R.5
  • 38
    • 84871922836 scopus 로고    scopus 로고
    • Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses
    • Ciesielska, A., P. Hadaczek, G. Mittermeyer, et al., Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther. 21: p. 158–66.
    • Mol Ther , vol.21 , pp. 158-166
    • Ciesielska, A.1    Hadaczek, P.2    Mittermeyer, G.3
  • 39
    • 28444442243 scopus 로고    scopus 로고
    • Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
    • PID: 16226919, COI: 1:CAS:528:DC%2BD2MXht1Ghsb%2FO
    • Acland GM, Aguirre GD, Bennett J, et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther 2005;12:1072–1082.
    • (2005) Mol Ther , vol.12 , pp. 1072-1082
    • Acland, G.M.1    Aguirre, G.D.2    Bennett, J.3
  • 40
    • 0035032662 scopus 로고    scopus 로고
    • Gene therapy restores vision in a canine model of childhood blindness
    • PID: 11326284, COI: 1:CAS:528:DC%2BD3MXjt1ajsr0%3D
    • Acland GM, Aguirre GD, Ray J, et al. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 2001;28:92–95.
    • (2001) Nat Genet , vol.28 , pp. 92-95
    • Acland, G.M.1    Aguirre, G.D.2    Ray, J.3
  • 41
    • 77649242176 scopus 로고    scopus 로고
    • Gene therapy for Leber’s congenital amaurosis is safe and effective through 1.5 years after vector administration
    • PID: 19953081, COI: 1:CAS:528:DC%2BD1MXhsV2ktrbO
    • Simonelli F, Maguire AM, Testa F, et al. Gene therapy for Leber’s congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 2010;18:643–650.
    • (2010) Mol Ther , vol.18 , pp. 643-650
    • Simonelli, F.1    Maguire, A.M.2    Testa, F.3
  • 42
    • 70350620424 scopus 로고    scopus 로고
    • Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial
    • PID: 19854499, COI: 1:CAS:528:DC%2BD1MXhtlyjtLrK
    • Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009;374:1597–1605.
    • (2009) Lancet , vol.374 , pp. 1597-1605
    • Maguire, A.M.1    High, K.A.2    Auricchio, A.3
  • 43
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • PID: 18688285, COI: 1:CAS:528:DC%2BD1cXhtV2isbrK
    • Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 2008;118:3132–3142.
    • (2008) J Clin Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1    Garrigue, A.2    Wang, G.P.3
  • 44
    • 0142089747 scopus 로고    scopus 로고
    • Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
    • PID: 14567964, COI: 1:CAS:528:DC%2BD3sXotVChtrw%3D
    • Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80:148–158.
    • (2003) Mol Genet Metab , vol.80 , pp. 148-158
    • Raper, S.E.1    Chirmule, N.2    Lee, F.S.3
  • 45
    • 84866899464 scopus 로고    scopus 로고
    • Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union
    • PID: 23023051
    • Ylä-Herttuala S. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Mol Ther 2012;20:1831–1832.
    • (2012) Mol Ther , vol.20 , pp. 1831-1832
    • Ylä-Herttuala, S.1
  • 46
    • 84855814958 scopus 로고    scopus 로고
    • Non-viral gene therapy for neurological diseases, with an emphasis on targeted gene delivery
    • PID: 21893121, COI: 1:CAS:528:DC%2BC38Xnt1WisA%3D%3D
    • Rogers ML, Rush RA. Non-viral gene therapy for neurological diseases, with an emphasis on targeted gene delivery. J Control Release 2012;157:183–189.
    • (2012) J Control Release , vol.157 , pp. 183-189
    • Rogers, M.L.1    Rush, R.A.2
  • 47
    • 84860171925 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
    • PID: 22201473, COI: 1:CAS:528:DC%2BC38XlvVGis74%3D
    • Samaranch L, Salegio EA, San Sebastian W, et al. Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther 2012;23:382–389.
    • (2012) Hum Gene Ther , vol.23 , pp. 382-389
    • Samaranch, L.1    Salegio, E.A.2    San Sebastian, W.3
  • 48
    • 79957888016 scopus 로고    scopus 로고
    • Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates
    • PID: 21487395, COI: 1:CAS:528:DC%2BC3MXks1agsrc%3D
    • Gray SJ, Matagne V, Bachaboina L, et al. Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther 2011;19:1058–1069.
    • (2011) Mol Ther , vol.19 , pp. 1058-1069
    • Gray, S.J.1    Matagne, V.2    Bachaboina, L.3
  • 49
    • 79961032418 scopus 로고    scopus 로고
    • Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    • PID: 21610699, COI: 1:CAS:528:DC%2BC3MXmsVGmtLY%3D
    • Zhang H, Yang B, Mu X, et al. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 2011;19:1440–1448.
    • (2011) Mol Ther , vol.19 , pp. 1440-1448
    • Zhang, H.1    Yang, B.2    Mu, X.3
  • 51
    • 84856689838 scopus 로고    scopus 로고
    • Oncolytic virus therapy for glioblastoma multiforme: concepts and candidates
    • PID: 22290260, COI: 1:CAS:528:DC%2BC38XktFCmtLc%3D
    • Wollmann G, Ozduman K, van den Pol AN. Oncolytic virus therapy for glioblastoma multiforme: concepts and candidates. Cancer J 2012;18:69–81.
    • (2012) Cancer J , vol.18 , pp. 69-81
    • Wollmann, G.1    Ozduman, K.2    van den Pol, A.N.3
  • 52
    • 0038288854 scopus 로고    scopus 로고
    • Preclinical characterization of the antiglioma activity of a tropism-enhanced adenovirus targeted to the retinoblastoma pathway
    • PID: 12734316, COI: 1:CAS:528:DC%2BD3sXjslGrsLo%3D
    • Fueyo J, Alemany R, Gomez-Manzano V, et al. Preclinical characterization of the antiglioma activity of a tropism-enhanced adenovirus targeted to the retinoblastoma pathway. J Natl Cancer Inst 2003;95:652–660.
    • (2003) J Natl Cancer Inst , vol.95 , pp. 652-660
    • Fueyo, J.1    Alemany, R.2    Gomez-Manzano, V.3
  • 53
    • 13844254649 scopus 로고    scopus 로고
    • Rescue and propagation of fully retargeted oncolytic measles viruses
    • PID: 15685166, COI: 1:CAS:528:DC%2BD2MXhtVGnu7o%3D
    • Nakamura T, Peng KW, Harvey M, et al. Rescue and propagation of fully retargeted oncolytic measles viruses. Nat Biotechnol 2005;23:209–214.
    • (2005) Nat Biotechnol , vol.23 , pp. 209-214
    • Nakamura, T.1    Peng, K.W.2    Harvey, M.3
  • 54
    • 84857569415 scopus 로고    scopus 로고
    • Brain tumor eradication and prolonged survival from intratumoral conversion of 5-fluorocytosine to 5-fluorouracil using a nonlytic retroviral replicating vector
    • Ostertag D, Amundson KK, Lopez Espinoza F, et al. Brain tumor eradication and prolonged survival from intratumoral conversion of 5-fluorocytosine to 5-fluorouracil using a nonlytic retroviral replicating vector. Neuro Oncol 14:145–159.
    • Neuro Oncol , vol.14 , pp. 145-159
    • Ostertag, D.1    Amundson, K.K.2    Lopez Espinoza, F.3
  • 55
    • 0028050350 scopus 로고
    • Rapid evolution of a protein in vitro by DNA shuffling
    • PID: 8047147, COI: 1:CAS:528:DyaK2cXlvFOjsrs%3D
    • Stemmer WP. Rapid evolution of a protein in vitro by DNA shuffling. Nature 1994;370:389–391.
    • (1994) Nature , vol.370 , pp. 389-391
    • Stemmer, W.P.1
  • 56
    • 45749099260 scopus 로고    scopus 로고
    • Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles
    • PID: 18500254, COI: 1:CAS:528:DC%2BD1cXns1eiurs%3D
    • Li W, Asokan A, Wu Z, et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther 2008;16:1252–1260.
    • (2008) Mol Ther , vol.16 , pp. 1252-1260
    • Li, W.1    Asokan, A.2    Wu, Z.3
  • 57
    • 44949131860 scopus 로고    scopus 로고
    • In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
    • PID: 18400866, COI: 1:CAS:528:DC%2BD1cXnt1Chur8%3D
    • Grimm D, Lee JS, Wang L, et al. In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol 2008;82:5887–5911.
    • (2008) J Virol , vol.82 , pp. 5887-5911
    • Grimm, D.1    Lee, J.S.2    Wang, L.3
  • 58
    • 54849391500 scopus 로고    scopus 로고
    • DNA shuffling of adeno-associated virus yields functionally diverse viral progeny
    • PID: 18728640, COI: 1:CAS:528:DC%2BD1cXhtFGhs7bK
    • Koerber JT, Jang JH, Schaffer DV. DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther 2008;16:1703–1709.
    • (2008) Mol Ther , vol.16 , pp. 1703-1709
    • Koerber, J.T.1    Jang, J.H.2    Schaffer, D.V.3
  • 59
    • 0033675531 scopus 로고    scopus 로고
    • Breeding of retroviruses by DNA shuffling for improved stability and processing yields
    • PID: 11101807, COI: 1:CAS:528:DC%2BD3cXovVOgsb0%3D
    • Powell SK, Kaloss MA, Pinkstaff A, et al. Breeding of retroviruses by DNA shuffling for improved stability and processing yields. Nat Biotechnol 2000;18:1279–1282.
    • (2000) Nat Biotechnol , vol.18 , pp. 1279-1282
    • Powell, S.K.1    Kaloss, M.A.2    Pinkstaff, A.3
  • 60
    • 75049084578 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus for glioma cell transduction
    • PID: 19618115
    • Maguire CA, Gianni D, Meijer DH, et al. Directed evolution of adeno-associated virus for glioma cell transduction. J Neurooncol 2010;96:337–347.
    • (2010) J Neurooncol , vol.96 , pp. 337-347
    • Maguire, C.A.1    Gianni, D.2    Meijer, D.H.3
  • 61
    • 77649273563 scopus 로고    scopus 로고
    • Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB)
    • PID: 20040913, COI: 1:CAS:528:DC%2BD1MXhs1WntrrN
    • Gray SJ, Blake BL, Criswell HE, et al. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther 2010;18:570–578.
    • (2010) Mol Ther , vol.18 , pp. 570-578
    • Gray, S.J.1    Blake, B.L.2    Criswell, H.E.3
  • 62
    • 73849091644 scopus 로고    scopus 로고
    • Molecular evolution of adeno-associated virus for enhanced glial gene delivery
    • PID: 19672246, COI: 1:CAS:528:DC%2BD1MXps1ahtLo%3D
    • Koerber JT, Klimczak R, Jang JH, et al. Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol Ther 2009;17:2088–2095.
    • (2009) Mol Ther , vol.17 , pp. 2088-2095
    • Koerber, J.T.1    Klimczak, R.2    Jang, J.H.3
  • 63
    • 79953325690 scopus 로고    scopus 로고
    • An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells
    • PID: 21224831, COI: 1:CAS:528:DC%2BC3MXjslWqtA%3D%3D
    • Jang JH, Koerber JT, Kim JS, et al. An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells. Mol Ther 2011;19:667–675.
    • (2011) Mol Ther , vol.19 , pp. 667-675
    • Jang, J.H.1    Koerber, J.T.2    Kim, J.S.3
  • 64
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
    • PID: 16429148, COI: 1:CAS:528:DC%2BD28XhtFGqs74%3D
    • Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 2006;24:198–204.
    • (2006) Nat Biotechnol , vol.24 , pp. 198-204
    • Maheshri, N.1    Koerber, J.T.2    Kaspar, B.K.3    Schaffer, D.V.4
  • 65
    • 84890401599 scopus 로고    scopus 로고
    • Current progress on aptamer-targeted oligonucleotide therapeutics
    • PID: 24304250, COI: 1:CAS:528:DC%2BC3sXhvVyitbvI
    • Dassie JP, Giangrande PH. Current progress on aptamer-targeted oligonucleotide therapeutics. Ther Deliv 2013;4:1527–1546.
    • (2013) Ther Deliv , vol.4 , pp. 1527-1546
    • Dassie, J.P.1    Giangrande, P.H.2
  • 66
    • 78049345098 scopus 로고    scopus 로고
    • Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors
    • PID: 20935652, COI: 1:CAS:528:DC%2BC3cXht1Kms7jJ
    • Anliker B, Abel T, Kneissl S, et al. Specific gene transfer to neurons, endothelial cells and hematopoietic progenitors with lentiviral vectors. Nat Methods 2010;7:929–935.
    • (2010) Nat Methods , vol.7 , pp. 929-935
    • Anliker, B.1    Abel, T.2    Kneissl, S.3
  • 67
    • 78650537312 scopus 로고    scopus 로고
    • Gene therapy-mediated delivery of targeted cytotoxins for glioma therapeutics
    • PID: 21030678, COI: 1:CAS:528:DC%2BC3cXhsVyitLvK
    • Candolfi M, Xiong W, Yagiz K, et al. Gene therapy-mediated delivery of targeted cytotoxins for glioma therapeutics. Proc Natl Acad Sci USA 2010;107:20021–20026.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 20021-20026
    • Candolfi, M.1    Xiong, W.2    Yagiz, K.3
  • 68
    • 84875215479 scopus 로고    scopus 로고
    • Effective treatment of an orthotopic xenograft model of human glioblastoma using an EGFR-retargeted oncolytic herpes simplex virus
    • PID: 23070115, COI: 1:CAS:528:DC%2BC38XhsFWqsr7L
    • Uchida H, Marzulli M, Nakano K, et al. Effective treatment of an orthotopic xenograft model of human glioblastoma using an EGFR-retargeted oncolytic herpes simplex virus. Mol Ther 2013;21:561–569.
    • (2013) Mol Ther , vol.21 , pp. 561-569
    • Uchida, H.1    Marzulli, M.2    Nakano, K.3
  • 69
    • 54749120988 scopus 로고    scopus 로고
    • Preventing growth of brain tumors by creating a zone of resistance
    • PID: 18714312, COI: 1:CAS:528:DC%2BD1cXhtFGhs7nK
    • Maguire CA, Meijer DH, LeRoy SG, et al. Preventing growth of brain tumors by creating a zone of resistance. Mol Ther 2008;16:1695–1702.
    • (2008) Mol Ther , vol.16 , pp. 1695-1702
    • Maguire, C.A.1    Meijer, D.H.2    LeRoy, S.G.3
  • 70
    • 0037297303 scopus 로고    scopus 로고
    • Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area
    • PID: 12595892, COI: 1:STN:280:DC%2BD3s%2Fotl2qsw%3D%3D
    • Kugler S, Kilic E, Bahr M. Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area. Gene Ther 2003;10:337–347.
    • (2003) Gene Ther , vol.10 , pp. 337-347
    • Kugler, S.1    Kilic, E.2    Bahr, M.3
  • 71
    • 33749233855 scopus 로고    scopus 로고
    • Astrocytic expression of transgene in the rat brain mediated by baculovirus vectors containing an astrocyte-specific promoter
    • PID: 16724097, COI: 1:CAS:528:DC%2BD28XhtVans7zE
    • Wang CY, Wang S. Astrocytic expression of transgene in the rat brain mediated by baculovirus vectors containing an astrocyte-specific promoter. Gene Ther 2006;13:1447–1456.
    • (2006) Gene Ther , vol.13 , pp. 1447-1456
    • Wang, C.Y.1    Wang, S.2
  • 72
    • 77955629913 scopus 로고    scopus 로고
    • Expression characteristics of dual-promoter lentiviral vectors targeting retinal photoreceptors and Muller cells
    • PID: 20517486, COI: 1:CAS:528:DC%2BC3cXntVGqtbc%3D
    • Semple-Rowland SL, Coggin WE, Geesey M, et al. Expression characteristics of dual-promoter lentiviral vectors targeting retinal photoreceptors and Muller cells. Mol Vis 2010;16:916–934.
    • (2010) Mol Vis , vol.16 , pp. 916-934
    • Semple-Rowland, S.L.1    Coggin, W.E.2    Geesey, M.3
  • 73
    • 78449270317 scopus 로고    scopus 로고
    • Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
    • Gentner B, Visigalli I, Hiramatsu H, et al. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2010;2:58–84.
    • (2010) Sci Transl Med , vol.2 , pp. 58-84
    • Gentner, B.1    Visigalli, I.2    Hiramatsu, H.3
  • 74
    • 84890120669 scopus 로고    scopus 로고
    • A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS gene therapy in canavan mice
    • PID: 23817205, COI: 1:CAS:528:DC%2BC3sXhtVehsLvK
    • Ahmed SS, Li H, Cao C, et al. A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS gene therapy in canavan mice. Mol Ther 2013; 21:2136–2147.
    • (2013) Mol Ther , vol.21 , pp. 2136-2147
    • Ahmed, S.S.1    Li, H.2    Cao, C.3
  • 75
    • 73949157699 scopus 로고    scopus 로고
    • In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance
    • Annoni A, Brown BD, Cantore A, et al. In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. Blood 2009;114:1552–1561.
    • (2009) Blood , vol.114 , pp. 1552-1561
    • Annoni, A.1    Brown, B.D.2    Cantore, A.3
  • 76
    • 84872226609 scopus 로고    scopus 로고
    • Safety profile of gutless adenovirus vectors delivered into the normal brain parenchyma: implications for a glioma phase 1 clinical trial
    • PID: 22950971
    • Muhammad AK, Xiong W, Puntel M, et al. Safety profile of gutless adenovirus vectors delivered into the normal brain parenchyma: implications for a glioma phase 1 clinical trial. Hum Gene Ther Methods 2012;23:271–284.
    • (2012) Hum Gene Ther Methods , vol.23 , pp. 271-284
    • Muhammad, A.K.1    Xiong, W.2    Puntel, M.3
  • 77
    • 84891797936 scopus 로고    scopus 로고
    • Lipid nanoparticle delivery of siRNA to silence neuronal gene expression in the brain
    • PID: 24301867, COI: 1:CAS:528:DC%2BC2cXkvVKiu78%3D
    • Rungta RL, Choi HB, Lin PJ, et al. Lipid nanoparticle delivery of siRNA to silence neuronal gene expression in the brain. Mol Ther Nucleic Acids 2013;2: e136.
    • (2013) Mol Ther Nucleic Acids , vol.2 , pp. e136
    • Rungta, R.L.1    Choi, H.B.2    Lin, P.J.3
  • 78
    • 51349102460 scopus 로고    scopus 로고
    • Antitumour activity of ANG1005, a conjugate between paclitaxel and the new brain delivery vector Angiopep-2
    • PID: 18574456, COI: 1:CAS:528:DC%2BD1cXhtVygtL3N
    • Regina A, Demeule M, Che C, et al. Antitumour activity of ANG1005, a conjugate between paclitaxel and the new brain delivery vector Angiopep-2. Br J Pharmacol 2008;155:185–197.
    • (2008) Br J Pharmacol , vol.155 , pp. 185-197
    • Regina, A.1    Demeule, M.2    Che, C.3
  • 79
    • 40849085422 scopus 로고    scopus 로고
    • Identification and design of peptides as a new drug delivery system for the brain
    • PID: 18156463, COI: 1:CAS:528:DC%2BD1cXivVajsL0%3D
    • Demeule M, Regina A, Che C, et al. Identification and design of peptides as a new drug delivery system for the brain. J Pharmacol Exp Ther 2008;324:1064–1072.
    • (2008) J Pharmacol Exp Ther , vol.324 , pp. 1064-1072
    • Demeule, M.1    Regina, A.2    Che, C.3
  • 80
    • 84883889734 scopus 로고    scopus 로고
    • Angiopep-conjugated nanoparticles for targeted long-term gene therapy of Parkinson’s disease
    • PID: 23703371, COI: 1:CAS:528:DC%2BC3sXot1KitL4%3D
    • Huang R, Ma H, Guo Y, et al. Angiopep-conjugated nanoparticles for targeted long-term gene therapy of Parkinson’s disease. Pharm Res 2013;30:2549–2559.
    • (2013) Pharm Res , vol.30 , pp. 2549-2559
    • Huang, R.1    Ma, H.2    Guo, Y.3
  • 81
    • 77957013817 scopus 로고    scopus 로고
    • Magnetic resonance monitoring of focused ultrasound/magnetic nanoparticle targeting delivery of therapeutic agents to the brain
    • PID: 20696897, COI: 1:CAS:528:DC%2BC3cXhtFSnsLnL
    • Liu HL, Hua MY, Yang HW, et al. Magnetic resonance monitoring of focused ultrasound/magnetic nanoparticle targeting delivery of therapeutic agents to the brain. Proc Natl Acad Sci USA 2010;107:15205–15210.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 15205-15210
    • Liu, H.L.1    Hua, M.Y.2    Yang, H.W.3
  • 82
    • 84861882656 scopus 로고    scopus 로고
    • Effective gene transfer into central nervous system following ultrasound-microbubbles-induced opening of the blood-brain barrier
    • PID: 22677255
    • Huang, Q, Deng J, Xie Z, et al. Effective gene transfer into central nervous system following ultrasound-microbubbles-induced opening of the blood-brain barrier. Ultrasound Med Biol 2012;38:1234–1243.
    • (2012) Ultrasound Med Biol , vol.38 , pp. 1234-1243
    • Huang, Q.1    Deng, J.2    Xie, Z.3
  • 83
    • 85027935991 scopus 로고    scopus 로고
    • Brain tumor microvesicles: insights into intercellular communication in the nervous system
    • PID: 21553248, COI: 1:CAS:528:DC%2BC3MXps1Wit7o%3D
    • van der Vos KE, Balaj L, Skog J, Breakefield XO. Brain tumor microvesicles: insights into intercellular communication in the nervous system. Cell Mol Neurobiol 2011;31:949–959.
    • (2011) Cell Mol Neurobiol , vol.31 , pp. 949-959
    • van der Vos, K.E.1    Balaj, L.2    Skog, J.3    Breakefield, X.O.4
  • 84
    • 79953858598 scopus 로고    scopus 로고
    • Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes
    • PID: 21423189, COI: 1:CAS:528:DC%2BC3MXjsVKqsLw%3D
    • Alvarez-Erviti L, Seow Y, Yin H, et al. Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol 2011;29:341–345.
    • (2011) Nat Biotechnol , vol.29 , pp. 341-345
    • Alvarez-Erviti, L.1    Seow, Y.2    Yin, H.3
  • 85
    • 84884187644 scopus 로고    scopus 로고
    • Electroporation-induced siRNA precipitation obscures the efficiency of siRNA loading into extracellular vesicles
    • PID: 23994516, COI: 1:CAS:528:DC%2BC3sXhslOktLjL
    • Kooijmans SA, Stremersch S, Braeckmans K, et al. Electroporation-induced siRNA precipitation obscures the efficiency of siRNA loading into extracellular vesicles. J Control Release 2013;172:229–238.
    • (2013) J Control Release , vol.172 , pp. 229-238
    • Kooijmans, S.A.1    Stremersch, S.2    Braeckmans, K.3
  • 86
    • 84902544740 scopus 로고    scopus 로고
    • Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo
    • PID: 24917028, COI: 1:CAS:528:DC%2BC2cXps1yrsrw%3D
    • Gyorgy B, Fitzpatrick Z, Crommentuijn MH et al. Naturally enveloped AAV vectors for shielding neutralizing antibodies and robust gene delivery in vivo. Biomaterials 2014;35:7598–7560.
    • (2014) Biomaterials , vol.35 , pp. 7598-7560
    • Gyorgy, B.1    Fitzpatrick, Z.2    Crommentuijn, M.H.3
  • 87
    • 57049103401 scopus 로고    scopus 로고
    • Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers
    • PID: 19011622, COI: 1:CAS:528:DC%2BD1cXhsVentbfI
    • Skog J, Wurdinger T, van Rijn S, et al. Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers. Nat Cell Biol 2008;10:1470–1476.
    • (2008) Nat Cell Biol , vol.10 , pp. 1470-1476
    • Skog, J.1    Wurdinger, T.2    van Rijn, S.3
  • 88
    • 84890907806 scopus 로고    scopus 로고
    • Sumoylated hnRNPA2B1 controls the sorting of miRNAs into exosomes through binding to specific motifs
    • PID: 24356509
    • Villarroya-Beltri C, Gutierrez-Vazquez C, Sanchez-Cabo F, et al. Sumoylated hnRNPA2B1 controls the sorting of miRNAs into exosomes through binding to specific motifs. Nat Commun 2013;4:2980.
    • (2013) Nat Commun , vol.4 , pp. 2980
    • Villarroya-Beltri, C.1    Gutierrez-Vazquez, C.2    Sanchez-Cabo, F.3
  • 89
    • 84866364616 scopus 로고    scopus 로고
    • miR-1289 and “zipcode”-like sequence enrich mRNAs in microvesicles
    • PID: 23344721
    • Bolukbasi MF, Mizrak A, Ozdener GB, et al. miR-1289 and “zipcode”-like sequence enrich mRNAs in microvesicles. Mol Ther Nucleic Acids 2012;1:e10.
    • (2012) Mol Ther Nucleic Acids , vol.1 , pp. e10
    • Bolukbasi, M.F.1    Mizrak, A.2    Ozdener, G.B.3
  • 90
    • 84886625070 scopus 로고    scopus 로고
    • Delivery of functional anti-miR-9 by mesenchymal stem cell-derived exosomes to glioblastoma multiforme cells conferred chemosensitivity
    • PID: 24084846
    • Munoz JL, Bliss SA, Greco SJ, et al. Delivery of functional anti-miR-9 by mesenchymal stem cell-derived exosomes to glioblastoma multiforme cells conferred chemosensitivity. Mol Ther Nucleic Acids 2013;2:e126.
    • (2013) Mol Ther Nucleic Acids , vol.2 , pp. e126
    • Munoz, J.L.1    Bliss, S.A.2    Greco, S.J.3
  • 91
    • 84896896916 scopus 로고    scopus 로고
    • Neural stem cells improve intracranial nanoparticle retention and tumor-selective distribution
    • PID: 24559447, COI: 1:CAS:528:DC%2BC2cXivFelsr8%3D
    • Mooney R, Weng Y, Tirughana-Sambandan R, et al. Neural stem cells improve intracranial nanoparticle retention and tumor-selective distribution. Future Oncol 2014;10:401–415.
    • (2014) Future Oncol , vol.10 , pp. 401-415
    • Mooney, R.1    Weng, Y.2    Tirughana-Sambandan, R.3
  • 92
    • 84894515770 scopus 로고    scopus 로고
    • Transplants of adult mesenchymal and neural stem cells provide neuroprotection and behavioral sparing in a transgenic rat model of Huntington’s disease
    • PID: 23939879, COI: 1:CAS:528:DC%2BC2cXlsFOrur8%3D
    • Rossignol J, Fink K, Davis K, et al. Transplants of adult mesenchymal and neural stem cells provide neuroprotection and behavioral sparing in a transgenic rat model of Huntington’s disease. Stem Cells 2014;32:500–509.
    • (2014) Stem Cells , vol.32 , pp. 500-509
    • Rossignol, J.1    Fink, K.2    Davis, K.3
  • 93
    • 33646034578 scopus 로고    scopus 로고
    • A hybrid vector for ligand-directed tumor targeting and molecular imaging
    • PID: 16630824, COI: 1:CAS:528:DC%2BD28Xkt1Oqurs%3D
    • Hajitou A, Trepel M, Lilley CE, et al. A hybrid vector for ligand-directed tumor targeting and molecular imaging. Cell 2006;125:385–398.
    • (2006) Cell , vol.125 , pp. 385-398
    • Hajitou, A.1    Trepel, M.2    Lilley, C.E.3
  • 94
    • 84871389700 scopus 로고    scopus 로고
    • Dual systemic tumor targeting with ligand-directed phage and Grp78 promoter induces tumor regression
    • PID: 23053496, COI: 1:CAS:528:DC%2BC38XhvVSqt7jI
    • Kia A, Przystal JM, Nianiaris N, et al. Dual systemic tumor targeting with ligand-directed phage and Grp78 promoter induces tumor regression. Mol Cancer Ther 2012;11:2566–2577.
    • (2012) Mol Cancer Ther , vol.11 , pp. 2566-2577
    • Kia, A.1    Przystal, J.M.2    Nianiaris, N.3
  • 95
    • 84860526022 scopus 로고    scopus 로고
    • Microvesicle-associated AAV vector as a novel gene delivery system
    • PID: 22314290, COI: 1:CAS:528:DC%2BC38XhvVGlt70%3D
    • Maguire CA, Balaj L, Sivaraman S, et al. Microvesicle-associated AAV vector as a novel gene delivery system. Mol Ther 2012;20:960–971.
    • (2012) Mol Ther , vol.20 , pp. 960-971
    • Maguire, C.A.1    Balaj, L.2    Sivaraman, S.3
  • 96
    • 80455173951 scopus 로고    scopus 로고
    • Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders
    • PID: 21811247, COI: 1:CAS:528:DC%2BC3MXpslShu7o%3D
    • Bevan AK, Duque S, Foust KD, et al. Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther 2011;19:1971–1980.
    • (2011) Mol Ther , vol.19 , pp. 1971-1980
    • Bevan, A.K.1    Duque, S.2    Foust, K.D.3
  • 97
    • 84903760379 scopus 로고    scopus 로고
    • Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10
    • PID: 24781136, COI: 1:CAS:528:DC%2BC2cXhtVamu7rP
    • Yang B, Li S, Wang H, et al. Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther 2014; 22:1299–1309.
    • (2014) Mol Ther , vol.22 , pp. 1299-1309
    • Yang, B.1    Li, S.2    Wang, H.3
  • 98
    • 67649861393 scopus 로고    scopus 로고
    • Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
    • PID: 19367261, COI: 1:CAS:528:DC%2BD1MXksVyis7Y%3D
    • Duque S, Joussemet B, Riviere C, et al. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther 2009;17:1187–1196.
    • (2009) Mol Ther , vol.17 , pp. 1187-1196
    • Duque, S.1    Joussemet, B.2    Riviere, C.3
  • 99
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • PID: 15294177, COI: 1:CAS:528:DC%2BD2cXmt1Cms7g%3D
    • Burger C, Gorbatyuk OS, Velardo MJ, et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 2004;10:302–317.
    • (2004) Mol Ther , vol.10 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3
  • 100
    • 54749126318 scopus 로고    scopus 로고
    • Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
    • PID: 18714307, COI: 1:CAS:528:DC%2BD1cXhtFGhs7bN
    • Cearley CN, Vandenberghe LH, Parente MK, et al. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther 2008;16:1710–1718.
    • (2008) Mol Ther , vol.16 , pp. 1710-1718
    • Cearley, C.N.1    Vandenberghe, L.H.2    Parente, M.K.3
  • 101
    • 32944459983 scopus 로고    scopus 로고
    • Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
    • PID: 16413228, COI: 1:CAS:528:DC%2BD28Xhslykt78%3D
    • Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther 2006;13:528–537.
    • (2006) Mol Ther , vol.13 , pp. 528-537
    • Cearley, C.N.1    Wolfe, J.H.2
  • 102
    • 70349850388 scopus 로고    scopus 로고
    • Young, During MJ. Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
    • PID: 19638961, COI: 1:CAS:528:DC%2BD1MXptFGru7o%3D
    • Lawlor PA, Bland RJ, Mouravlev A, Young, During MJ. Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol Ther 2009;17:1692–1702.
    • (2009) Mol Ther , vol.17 , pp. 1692-1702
    • Lawlor, P.A.1    Bland, R.J.2    Mouravlev, A.3
  • 103
    • 84890415169 scopus 로고    scopus 로고
    • Characterization of the properties of seven promoters in the motor cortex of rats and monkeys after lentiviral vector-mediated gene transfer
    • PID: 23964981, COI: 1:CAS:528:DC%2BC3sXhvFalu7zI
    • Yaguchi M, Ohashi Y, Tsubota T, et al. Characterization of the properties of seven promoters in the motor cortex of rats and monkeys after lentiviral vector-mediated gene transfer. Hum Gene Ther Methods 2013;24:333–344.
    • (2013) Hum Gene Ther Methods , vol.24 , pp. 333-344
    • Yaguchi, M.1    Ohashi, Y.2    Tsubota, T.3
  • 104
    • 0024362504 scopus 로고
    • Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5
    • PID: 2551778, COI: 1:CAS:528:DyaL1MXltFShur4%3D
    • Miyazaki J, Takaki S, Araki K, et al. Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5. Gene 1989;79:269–277.
    • (1989) Gene , vol.79 , pp. 269-277
    • Miyazaki, J.1    Takaki, S.2    Araki, K.3
  • 105
    • 0025884056 scopus 로고
    • Efficient selection for high-expression transfectants with a novel eukaryotic vector
    • PID: 1660837, COI: 1:CAS:528:DyaK38Xlt1GksA%3D%3D
    • Niwa H, Yamamura K, Miyazaki J. Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 1991;108:193–199.
    • (1991) Gene , vol.108 , pp. 193-199
    • Niwa, H.1    Yamamura, K.2    Miyazaki, J.3
  • 106
    • 77649270699 scopus 로고    scopus 로고
    • Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates
    • PID: 19773746, COI: 1:CAS:528:DC%2BD1MXhtFGrsbzJ
    • Dodiya HB, Bjorklund T, Stansell J 3rd, et al. Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates. Mol Ther 2010;18:579–587.
    • (2010) Mol Ther , vol.18 , pp. 579-587
    • Dodiya, H.B.1    Bjorklund, T.2    Stansell, J.3
  • 107
    • 77649271981 scopus 로고    scopus 로고
    • Comparative transduction efficiency of AAV vector serotypes 1–6 in the substantia nigra and striatum of the primate brain
    • PID: 20010918, COI: 1:CAS:528:DC%2BD1MXhsFKjsr%2FN
    • Markakis EA, Vives KP, Bober J, et al. Comparative transduction efficiency of AAV vector serotypes 1–6 in the substantia nigra and striatum of the primate brain. Mol Ther 2010;18:588–593.
    • (2010) Mol Ther , vol.18 , pp. 588-593
    • Markakis, E.A.1    Vives, K.P.2    Bober, J.3
  • 108
    • 0038618702 scopus 로고    scopus 로고
    • Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice
    • PID: 12768022, COI: 1:CAS:528:DC%2BD3sXksFGhsL4%3D
    • Passini MA, Watson DJ, Vite CH, et al. Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice. J Virol 2003;77:7034–7040.
    • (2003) J Virol , vol.77 , pp. 7034-7040
    • Passini, M.A.1    Watson, D.J.2    Vite, C.H.3
  • 109
    • 0034724209 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system
    • PID: 10688913, COI: 1:CAS:528:DC%2BD3cXitlWqt70%3D
    • Davidson BL, Stein CS, Heth JA, et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci USA 2000;97:3428–3432.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 3428-3432
    • Davidson, B.L.1    Stein, C.S.2    Heth, J.A.3
  • 110
    • 84879041937 scopus 로고    scopus 로고
    • Glial promoter selectivity following AAV-delivery to the immature brain
    • von Jonquieres G, Mersmann N, Klugmann CB, et al. Glial promoter selectivity following AAV-delivery to the immature brain. PLoS One 2013;8:e65646.
    • (2013) PLoS One , vol.8 , pp. e65646
    • von Jonquieres, G.1    Mersmann, N.2    Klugmann, C.B.3
  • 111
    • 84893586080 scopus 로고    scopus 로고
    • Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord
    • PID: 24191919, COI: 1:CAS:528:DC%2BC2cXhvVyksL4%3D
    • Dirren E, Towne CL, Setola V, et al. Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord. Hum Gene Ther 2014;25:109–120.
    • (2014) Hum Gene Ther , vol.25 , pp. 109-120
    • Dirren, E.1    Towne, C.L.2    Setola, V.3
  • 112
    • 0033557611 scopus 로고    scopus 로고
    • Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus
    • PID: 10723060, COI: 1:CAS:528:DyaK1MXhtlWlu7w%3D
    • Chen H, McCarty DM, Bruce AT, Suzuki K. Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus. J Neurosci Res 1999;55:504–513.
    • (1999) J Neurosci Res , vol.55 , pp. 504-513
    • Chen, H.1    McCarty, D.M.2    Bruce, A.T.3    Suzuki, K.4
  • 113
    • 73449128979 scopus 로고    scopus 로고
    • Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
    • PID: 19828868, COI: 1:CAS:528:DC%2BD1MXhtlyrtLzJ
    • Christine CW, Starr PA, Larson PS, et al. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 2009;73:1662–1669.
    • (2009) Neurology , vol.73 , pp. 1662-1669
    • Christine, C.W.1    Starr, P.A.2    Larson, P.S.3
  • 114
    • 44949222522 scopus 로고    scopus 로고
    • Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
    • PID: 18401019, COI: 1:CAS:528:DC%2BD1cXlvVCmtro%3D
    • Eberling JL, Jagust WJ, Christine CW, et al. Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 2008;70:1980–1983.
    • (2008) Neurology , vol.70 , pp. 1980-1983
    • Eberling, J.L.1    Jagust, W.J.2    Christine, C.W.3
  • 115
    • 84861139370 scopus 로고    scopus 로고
    • Gene therapy for aromatic l-amino acid decarboxylase deficiency
    • PID: 22593174
    • Hwu WL, Muramatsu S, Tseng SH, et al. Gene therapy for aromatic l-amino acid decarboxylase deficiency. Sci Transl Med 2012;4:134ra61.
    • (2012) Sci Transl Med , vol.4 , pp. 134ra61
    • Hwu, W.L.1    Muramatsu, S.2    Tseng, S.H.3
  • 116
    • 77956262279 scopus 로고    scopus 로고
    • A phase I study of aromatic l-amino acid decarboxylase gene therapy for Parkinson’s disease
    • PID: 20606642, COI: 1:CAS:528:DC%2BC3cXosVKitL8%3D
    • Muramatsu S, Fujimoto K, Kato S, et al. A phase I study of aromatic l-amino acid decarboxylase gene therapy for Parkinson’s disease. Mol Ther 2010;18:1731–1735.
    • (2010) Mol Ther , vol.18 , pp. 1731-1735
    • Muramatsu, S.1    Fujimoto, K.2    Kato, S.3
  • 117
    • 85027921807 scopus 로고    scopus 로고
    • Rafii MS, Baumann TL, Bakay RA, et al. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease. Alzheimers Dement, 2014 Jan 7 [Epub ahead of print].
    • Rafii MS, Baumann TL, Bakay RA, et al. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease. Alzheimers Dement, 2014 Jan 7 [Epub ahead of print].
  • 118
    • 34250683023 scopus 로고    scopus 로고
    • Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial
    • PID: 17586305, COI: 1:CAS:528:DC%2BD2sXmvVKqtb4%3D
    • Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial. Lancet 2007;369:2097–2105.
    • (2007) Lancet , vol.369 , pp. 2097-2105
    • Kaplitt, M.G.1    Feigin, A.2    Tang, C.3
  • 119
    • 44349136847 scopus 로고    scopus 로고
    • Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
    • PID: 18473686, COI: 1:CAS:528:DC%2BD1cXmsVGlsrg%3D
    • Worgall S, Sondhi D, Hackett NR, et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 2008;19:463–474.
    • (2008) Hum Gene Ther , vol.19 , pp. 463-474
    • Worgall, S.1    Sondhi, D.2    Hackett, N.R.3
  • 120
    • 84902952909 scopus 로고    scopus 로고
    • Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial
    • PID: 24524415, COI: 1:CAS:528:DC%2BC2cXhtVWmsL%2FI
    • Tardieu M, Zerah M, Husson B, et al. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Hum Gene Ther 2014;25:506–516.
    • (2014) Hum Gene Ther , vol.25 , pp. 506-516
    • Tardieu, M.1    Zerah, M.2    Husson, B.3
  • 121
    • 84871525461 scopus 로고    scopus 로고
    • Long-term follow-up after gene therapy for canavan disease
    • PID: 23253610
    • Leone P, Shera D, McPhee SE et al. Long-term follow-up after gene therapy for canavan disease. Sci Transl Med 2012;4:165ra163.
    • (2012) Sci Transl Med , vol.4 , pp. 165ra163
    • Leone, P.1    Shera, D.2    McPhee, S.E.3
  • 122
    • 77955175906 scopus 로고    scopus 로고
    • Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC
    • PID: 20531394, COI: 1:CAS:528:DC%2BC3cXntFSmtrk%3D
    • Hadaczek P, Eberling JL, Pivirotto P, et al. Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. Mol Ther 2010;18:1458–1461.
    • (2010) Mol Ther , vol.18 , pp. 1458-1461
    • Hadaczek, P.1    Eberling, J.L.2    Pivirotto, P.3
  • 123
    • 34548759601 scopus 로고    scopus 로고
    • Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease
    • PID: 17637720, COI: 1:CAS:528:DC%2BD2sXhtVGmtrbL
    • Cabrera-Salazar MA, Roskelley EM, Bu J, et al. Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease. Mol Ther 2007;15:1782–1788.
    • (2007) Mol Ther , vol.15 , pp. 1782-1788
    • Cabrera-Salazar, M.A.1    Roskelley, E.M.2    Bu, J.3
  • 124
    • 33746833590 scopus 로고    scopus 로고
    • Gene therapy of the brain in the dog model of Hurler’s syndrome
    • PID: 16718701, COI: 1:CAS:528:DC%2BD28XovFykt7k%3D
    • Ciron C, Desmaris N, Colle MA, et al. Gene therapy of the brain in the dog model of Hurler’s syndrome. Ann Neurol 2006;60:204–213.
    • (2006) Ann Neurol , vol.60 , pp. 204-213
    • Ciron, C.1    Desmaris, N.2    Colle, M.A.3
  • 125
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • PID: 20190738, COI: 1:CAS:528:DC%2BC3cXisFelurs%3D
    • Foust KD, Wang X, McGovern VL, et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 2010;28:271–274.
    • (2010) Nat Biotechnol , vol.28 , pp. 271-274
    • Foust, K.D.1    Wang, X.2    McGovern, V.L.3
  • 126
    • 84899117278 scopus 로고    scopus 로고
    • Sustained normalization of neurological disease after intracranial gene therapy in a feline model
    • PID: 24718858
    • McCurdy VJ, Johnson AK, Gray-Edwards HL, et al. Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Sci Transl Med 2014;6:231ra48.
    • (2014) Sci Transl Med , vol.6 , pp. 231ra48
    • McCurdy, V.J.1    Johnson, A.K.2    Gray-Edwards, H.L.3
  • 127
    • 14844284635 scopus 로고    scopus 로고
    • Effective gene therapy for an inherited CNS disease in a large animal model
    • PID: 15732095, COI: 1:CAS:528:DC%2BD2MXivVymsbc%3D
    • Vite CH, McGowan JC, Niogi SN, et al. Effective gene therapy for an inherited CNS disease in a large animal model. Ann Neurol 2005;57:355–364.
    • (2005) Ann Neurol , vol.57 , pp. 355-364
    • Vite, C.H.1    McGowan, J.C.2    Niogi, S.N.3
  • 128
    • 79957882063 scopus 로고    scopus 로고
    • Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
    • PID: 21386820, COI: 1:CAS:528:DC%2BC3MXivVGgt7s%3D
    • Fu H, Dirosario J, Killedar S, Zaraspe K, McCarty DM. Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol Ther 2011;19:1025–1033.
    • (2011) Mol Ther , vol.19 , pp. 1025-1033
    • Fu, H.1    Dirosario, J.2    Killedar, S.3    Zaraspe, K.4    McCarty, D.M.5
  • 129
    • 84902676801 scopus 로고    scopus 로고
    • Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
    • PID: 24720466, COI: 1:CAS:528:DC%2BC2cXpvVWrsL8%3D
    • Murrey DA, Naughton BJ, Duncan FJ, et al. Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Hum Gene Ther Clin Dev 2014;25:72–84.
    • (2014) Hum Gene Ther Clin Dev , vol.25 , pp. 72-84
    • Murrey, D.A.1    Naughton, B.J.2    Duncan, F.J.3
  • 130
    • 0030854815 scopus 로고    scopus 로고
    • Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase
    • PID: 9212105, COI: 1:CAS:528:DyaK2sXkt1Krtbk%3D
    • Taylor RM, Wolfe JH. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase. Nat Med 1997;3:771–774.
    • (1997) Nat Med , vol.3 , pp. 771-774
    • Taylor, R.M.1    Wolfe, J.H.2
  • 131
    • 60449089427 scopus 로고    scopus 로고
    • Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain
    • PID: 18818671, COI: 1:CAS:528:DC%2BD1MXhslSntr4%3D
    • Broekman ML, Tierney LA, Benn C, et al. Mechanisms of distribution of mouse beta-galactosidase in the adult GM1-gangliosidosis brain. Gene Ther 2009;16:303–308.
    • (2009) Gene Ther , vol.16 , pp. 303-308
    • Broekman, M.L.1    Tierney, L.A.2    Benn, C.3
  • 132
    • 0036703479 scopus 로고    scopus 로고
    • Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream
    • PID: 12151523, COI: 1:CAS:528:DC%2BD38XlvFKnsLs%3D
    • Passini MA, Lee EN, Heuer GG, Wolfe JH. Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J Neurosci 2002;22:6437–6446.
    • (2002) J Neurosci , vol.22 , pp. 6437-6446
    • Passini, M.A.1    Lee, E.N.2    Heuer, G.G.3    Wolfe, J.H.4
  • 133
    • 84864540429 scopus 로고    scopus 로고
    • Gene transfer corrects acute GM2 gangliosidosis—potential therapeutic contribution of perivascular enzyme flow
    • PID: 22453766, COI: 1:CAS:528:DC%2BC38Xkt1Gisr4%3D
    • Cachon-Gonzalez MB, Wang SZ, McNair R, et al. Gene transfer corrects acute GM2 gangliosidosis—potential therapeutic contribution of perivascular enzyme flow. Mol Ther 2012;20:1489–1500.
    • (2012) Mol Ther , vol.20 , pp. 1489-1500
    • Cachon-Gonzalez, M.B.1    Wang, S.Z.2    McNair, R.3
  • 134
    • 0037418258 scopus 로고    scopus 로고
    • Transgene produces massive overexpression of human beta-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors
    • PID: 12591953, COI: 1:CAS:528:DC%2BD3sXitVaitr8%3D
    • Vogler C, Galvin N, Levy B, et al. Transgene produces massive overexpression of human beta-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors. Proc Natl Acad Sci USA 2003;100:2669–2673.
    • (2003) Proc Natl Acad Sci USA , vol.100 , pp. 2669-2673
    • Vogler, C.1    Galvin, N.2    Levy, B.3
  • 135
    • 78149463784 scopus 로고    scopus 로고
    • AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival
    • PID: 20976108
    • Baek RC, Broekman ML, Leroy SG, et al. AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival. PLoS One 2010;5:e13468.
    • (2010) PLoS One , vol.5 , pp. e13468
    • Baek, R.C.1    Broekman, M.L.2    Leroy, S.G.3
  • 136
    • 84879693104 scopus 로고    scopus 로고
    • Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy
    • PID: 23689599, COI: 1:CAS:528:DC%2BC3sXnvV2rsb8%3D
    • Bradbury AM, Cochran JN, McCurdy VJ, et al. Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy. Mol Ther 2013;21:1306–1315.
    • (2013) Mol Ther , vol.21 , pp. 1306-1315
    • Bradbury, A.M.1    Cochran, J.N.2    McCurdy, V.J.3
  • 137
    • 84866984026 scopus 로고    scopus 로고
    • Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A
    • PID: 22828503, COI: 1:CAS:528:DC%2BC38XhtVOls7fI
    • Bu J, Ashe KM, Bringas J, et al. Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A. Mol Ther 2012;20:1893–1901.
    • (2012) Mol Ther , vol.20 , pp. 1893-1901
    • Bu, J.1    Ashe, K.M.2    Bringas, J.3
  • 138
    • 84865208842 scopus 로고    scopus 로고
    • Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression in the nonhuman primate brain
    • PID: 22574943, COI: 1:CAS:528:DC%2BC38Xht1ajsb3F
    • Salegio EA, Samaranch L, Jenkins RW, et al. Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression in the nonhuman primate brain. Hum Gene Ther 2012;23:891–902.
    • (2012) Hum Gene Ther , vol.23 , pp. 891-902
    • Salegio, E.A.1    Samaranch, L.2    Jenkins, R.W.3
  • 139
    • 84875443169 scopus 로고    scopus 로고
    • Long term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and non-human primates for the treatment of late infantile neuronal lipofuscinosis
    • PID: 23131032, COI: 1:CAS:528:DC%2BC3sXpvVOmtrk%3D
    • Sondhi D, Johnson L, De B, et al. Long term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and non-human primates for the treatment of late infantile neuronal lipofuscinosis. Hum Gene Ther Methods 2012;23:324–335.
    • (2012) Hum Gene Ther Methods , vol.23 , pp. 324-335
    • Sondhi, D.1    Johnson, L.2    De, B.3
  • 140
    • 84904412918 scopus 로고    scopus 로고
    • Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
    • PID: 24617515, COI: 1:CAS:528:DC%2BC2cXhtFKmurrF
    • Passini MA, Bu J, Richards AM, et al. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther 2014;25:619–630.
    • (2014) Hum Gene Ther , vol.25 , pp. 619-630
    • Passini, M.A.1    Bu, J.2    Richards, A.M.3
  • 141
    • 33749081269 scopus 로고    scopus 로고
    • Increased MECP2 gene copy number as the result of genomic duplication in neurodevelopmentally delayed males
    • PID: 17172942
    • del Gaudio D, Fang P, Scaglia F, et al. Increased MECP2 gene copy number as the result of genomic duplication in neurodevelopmentally delayed males. Genet Med 2006;8:784–792.
    • (2006) Genet Med , vol.8 , pp. 784-792
    • del Gaudio, D.1    Fang, P.2    Scaglia, F.3
  • 142
    • 33845772985 scopus 로고    scopus 로고
    • Recurrent infections, hypotonia, and mental retardation caused by duplication of MECP2 and adjacent region in Xq28
    • PID: 17088400
    • Friez MJ, Jones JR, Clarkson K, et al. Recurrent infections, hypotonia, and mental retardation caused by duplication of MECP2 and adjacent region in Xq28. Pediatrics 2006;118:e1687–e1695.
    • (2006) Pediatrics , vol.118 , pp. e1687-e1695
    • Friez, M.J.1    Jones, J.R.2    Clarkson, K.3
  • 143
    • 84871919786 scopus 로고    scopus 로고
    • Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice
    • PID: 23011033, COI: 1:CAS:528:DC%2BC38XhsVSlt7rI
    • Gadalla KK, Bailey ME, Spike RC, et al. Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. Mol Ther 2013;21:18–30.
    • (2013) Mol Ther , vol.21 , pp. 18-30
    • Gadalla, K.K.1    Bailey, M.E.2    Spike, R.C.3
  • 144
    • 84882718651 scopus 로고    scopus 로고
    • Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome
    • PID: 23966684, COI: 1:CAS:528:DC%2BC3sXhtlyisrbE
    • Garg SK, Lioy DT, Cheval H, et al. Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. J Neurosci 2013;33:13612–13620.
    • (2013) J Neurosci , vol.33 , pp. 13612-13620
    • Garg, S.K.1    Lioy, D.T.2    Cheval, H.3
  • 145
    • 28744458248 scopus 로고    scopus 로고
    • A segment of the Mecp2 promoter is sufficient to drive expression in neurons
    • PID: 16251199, COI: 1:CAS:528:DC%2BD2MXht1GgsrvE
    • Adachi M, Keefer EW, Jones FS. A segment of the Mecp2 promoter is sufficient to drive expression in neurons. Hum Mol Genet 2005;14:3709–3722.
    • (2005) Hum Mol Genet , vol.14 , pp. 3709-3722
    • Adachi, M.1    Keefer, E.W.2    Jones, F.S.3
  • 146
    • 0034882826 scopus 로고    scopus 로고
    • Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
    • PID: 11509958, COI: 1:CAS:528:DC%2BD3MXmtFensbk%3D
    • McCarty DM, Monahan PE, Samulski RJ. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 2001;8:1248–1254.
    • (2001) Gene Ther , vol.8 , pp. 1248-1254
    • McCarty, D.M.1    Monahan, P.E.2    Samulski, R.J.3
  • 147
    • 0346777307 scopus 로고    scopus 로고
    • Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    • PID: 14625565, COI: 1:CAS:528:DC%2BD3sXptVCkt74%3D
    • McCarty DM, Fu H, Monahan PE, et al. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003;10:2112–2118.
    • (2003) Gene Ther , vol.10 , pp. 2112-2118
    • McCarty, D.M.1    Fu, H.2    Monahan, P.E.3
  • 148
    • 80053009250 scopus 로고    scopus 로고
    • Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery
    • PID: 21443428, COI: 1:CAS:528:DC%2BC3MXhtF2rs7rL
    • Snyder BR, Gray SJ, Quach ET, et al. Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum Gene Ther 2011;22:1129–1135.
    • (2011) Hum Gene Ther , vol.22 , pp. 1129-1135
    • Snyder, B.R.1    Gray, S.J.2    Quach, E.T.3
  • 149
    • 80052982705 scopus 로고    scopus 로고
    • Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors
    • PID: 21476867, COI: 1:CAS:528:DC%2BC3MXhtF2rs7rP
    • Gray SJ, Foti SB, Schwartz JW, et al. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum Gene Ther 2011;22:1143–1153.
    • (2011) Hum Gene Ther , vol.22 , pp. 1143-1153
    • Gray, S.J.1    Foti, S.B.2    Schwartz, J.W.3
  • 150
    • 0028256226 scopus 로고
    • GFAP promoter directs astrocyte-specific expression in transgenic mice
    • PID: 8120611, COI: 1:CAS:528:DyaK2cXis1Kksr8%3D
    • Brenner M, Kisseberth WC, Su Y, Besnard F, Messing A. GFAP promoter directs astrocyte-specific expression in transgenic mice. J Neurosci 1994;14:1030–1037.
    • (1994) J Neurosci , vol.14 , pp. 1030-1037
    • Brenner, M.1    Kisseberth, W.C.2    Su, Y.3    Besnard, F.4    Messing, A.5
  • 151
    • 44449173870 scopus 로고    scopus 로고
    • GFAP promoter elements required for region-specific and astrocyte-specific expression
    • PID: 18240313
    • Lee Y, Messing A, Su M, Brenner M. GFAP promoter elements required for region-specific and astrocyte-specific expression. Glia 2008;56:481–493.
    • (2008) Glia , vol.56 , pp. 481-493
    • Lee, Y.1    Messing, A.2    Su, M.3    Brenner, M.4
  • 152
    • 17944378442 scopus 로고    scopus 로고
    • Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes
    • PID: 11571569, COI: 1:CAS:528:DC%2BD3MXmsFKlu78%3D
    • Xu R, Janson CG, Mastakov M, et al. Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes. Gene Ther 2001;8:1323–1332.
    • (2001) Gene Ther , vol.8 , pp. 1323-1332
    • Xu, R.1    Janson, C.G.2    Mastakov, M.3
  • 153
    • 84888324248 scopus 로고    scopus 로고
    • The microglial sensome revealed by direct RNA sequencing
    • PID: 24162652, COI: 1:CAS:528:DC%2BC3sXhs1yhsrnE
    • Hickman SE, Kingery ND, Ohsumi TK, et al. The microglial sensome revealed by direct RNA sequencing. Nat Neurosci 2013;16:1896–1905.
    • (2013) Nat Neurosci , vol.16 , pp. 1896-1905
    • Hickman, S.E.1    Kingery, N.D.2    Ohsumi, T.K.3
  • 154
    • 78049259570 scopus 로고    scopus 로고
    • A regulatory toolbox of MiniPromoters to drive selective expression in the brain
    • PID: 20807748, COI: 1:CAS:528:DC%2BC3cXht1WqsLjO
    • Portales-Casamar E, Swanson DJ, Liu L, et al. A regulatory toolbox of MiniPromoters to drive selective expression in the brain. Proc Natl Acad Sci USA 2010;107:16589–16594.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 16589-16594
    • Portales-Casamar, E.1    Swanson, D.J.2    Liu, L.3
  • 155
    • 85015620576 scopus 로고    scopus 로고
    • Targeted CNS delivery using human minipromoters and demonstrated compatibility with adeno-associated viral vectors
    • PID: 24761428
    • de Leeuw CN, Dyka FM, Boye SL, et al. Targeted CNS delivery using human minipromoters and demonstrated compatibility with adeno-associated viral vectors. Mol Ther Methods Clin Dev 2014;1:5.
    • (2014) Mol Ther Methods Clin Dev , vol.1 , pp. 5
    • de Leeuw, C.N.1    Dyka, F.M.2    Boye, S.L.3
  • 156
    • 78650679270 scopus 로고    scopus 로고
    • Transient neuronal inhibition reveals opposing roles of indirect and direct pathways in sensitization
    • PID: 21131952, COI: 1:CAS:528:DC%2BC3cXhsFajurfO
    • Ferguson SM, Eskenazi D, Ishikawa M, et al. Transient neuronal inhibition reveals opposing roles of indirect and direct pathways in sensitization. Nat Neurosci 2011;14:22–24.
    • (2011) Nat Neurosci , vol.14 , pp. 22-24
    • Ferguson, S.M.1    Eskenazi, D.2    Ishikawa, M.3
  • 157
    • 77953946912 scopus 로고    scopus 로고
    • Distinct roles of synaptic transmission in direct and indirect striatal pathways to reward and aversive behavior
    • PID: 20620875, COI: 1:CAS:528:DC%2BC3cXovFeiur8%3D
    • Hikida T, Kimura K, Wada N, Funabiki K, Nakanishi S. Distinct roles of synaptic transmission in direct and indirect striatal pathways to reward and aversive behavior. Neuron 2010;66:896–907.
    • (2010) Neuron , vol.66 , pp. 896-907
    • Hikida, T.1    Kimura, K.2    Wada, N.3    Funabiki, K.4    Nakanishi, S.5
  • 158
    • 0027412209 scopus 로고
    • Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter
    • PID: 7679117, COI: 1:CAS:528:DyaK3sXitVKhsbo%3D
    • Flotte TR, Afione SA, Solow R, et al. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter. J Biol Chem 1993;268:3781–3790.
    • (1993) J Biol Chem , vol.268 , pp. 3781-3790
    • Flotte, T.R.1    Afione, S.A.2    Solow, R.3
  • 159
    • 0033857632 scopus 로고    scopus 로고
    • Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element
    • PID: 10954575, COI: 1:CAS:528:DC%2BD3cXmsVantLg%3D
    • Haberman RP, McCown TJ, Samulski RJ. Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element. J Virol 2000;74:8732–8739.
    • (2000) J Virol , vol.74 , pp. 8732-8739
    • Haberman, R.P.1    McCown, T.J.2    Samulski, R.J.3
  • 160
    • 0034808550 scopus 로고    scopus 로고
    • A high-efficiency synthetic promoter that drives transgene expression selectively in noradrenergic neurons
    • PID: 11560767, COI: 1:CAS:528:DC%2BD3MXntFWiur4%3D
    • Hwang DY, Carlezon WA Jr, Isacson O, Kim KS. A high-efficiency synthetic promoter that drives transgene expression selectively in noradrenergic neurons. Hum Gene Ther 2001;12:1731–1740.
    • (2001) Hum Gene Ther , vol.12 , pp. 1731-1740
    • Hwang, D.Y.1    Carlezon, W.A.2    Isacson, O.3    Kim, K.S.4
  • 161
    • 84859513112 scopus 로고    scopus 로고
    • Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats
    • PID: 22173709, COI: 1:CAS:528:DC%2BC38Xlt1Sksb4%3D
    • Bruinstroop E, Cano G, Vanderhorst VG, et al. Spinal projections of the A5, A6 (locus coeruleus), and A7 noradrenergic cell groups in rats. J Comp Neurol 2012;520:1985–2001.
    • (2012) J Comp Neurol , vol.520 , pp. 1985-2001
    • Bruinstroop, E.1    Cano, G.2    Vanderhorst, V.G.3
  • 162
    • 33747368249 scopus 로고    scopus 로고
    • Blood-brain barrier: structural components and function under physiologic and pathologic conditions
    • PID: 18040800
    • Persidsky, Y., S.H. Ramirez, J. Haorah, and G.D. Kanmogne, Blood-brain barrier: structural components and function under physiologic and pathologic conditions. J Neuroimmune Pharmacol 2006;1:223–236.
    • (2006) J Neuroimmune Pharmacol , vol.1 , pp. 223-236
    • Persidsky, Y.1    Ramirez, S.H.2    Haorah, J.3    Kanmogne, G.D.4
  • 163
    • 29244463365 scopus 로고    scopus 로고
    • Astrocyte-endothelial interactions at the blood-brain barrier
    • PID: 16371949, COI: 1:CAS:528:DC%2BD2MXhtlaktbjP
    • Abbott NJ, Ronnback L, Hansson E. Astrocyte-endothelial interactions at the blood-brain barrier. Nat Rev Neurosci 2006;7:41–53.
    • (2006) Nat Rev Neurosci , vol.7 , pp. 41-53
    • Abbott, N.J.1    Ronnback, L.2    Hansson, E.3
  • 164
    • 77953687856 scopus 로고    scopus 로고
    • Regulation of P-glycoprotein and other ABC drug transporters at the blood-brain barrier
    • PID: 20417575, COI: 1:CAS:528:DC%2BC3cXntVeksLY%3D
    • Miller DS. Regulation of P-glycoprotein and other ABC drug transporters at the blood-brain barrier. Trends Pharmacol Sci 2010;31:246–254.
    • (2010) Trends Pharmacol Sci , vol.31 , pp. 246-254
    • Miller, D.S.1
  • 165
    • 76849112146 scopus 로고    scopus 로고
    • Nanotechnology applications for improved delivery of antiretroviral drugs to the brain
    • PID: 19914319, COI: 1:CAS:528:DC%2BC3cXis1emtr0%3D
    • Wong HL, Chattopadhyay N, Wu XY, Bendayan R. Nanotechnology applications for improved delivery of antiretroviral drugs to the brain. Adv Drug Deliv Rev 2010;62:503–517.
    • (2010) Adv Drug Deliv Rev , vol.62 , pp. 503-517
    • Wong, H.L.1    Chattopadhyay, N.2    Wu, X.Y.3    Bendayan, R.4
  • 166
    • 84871598987 scopus 로고    scopus 로고
    • Function of the blood-brain barrier and restriction of drug delivery to invasive glioma cells: findings in an orthotopic rat xenograft model of glioma
    • PID: 23014761, COI: 1:CAS:528:DC%2BC3sXlt1OjtA%3D%3D
    • Agarwal, S., P. Manchanda, M.A. Vogelbaum, J.R. Ohlfest, and W.F. Elmquist, Function of the blood-brain barrier and restriction of drug delivery to invasive glioma cells: findings in an orthotopic rat xenograft model of glioma. Drug Metab Dispos 2013;41:33–39.
    • (2013) Drug Metab Dispos , vol.41 , pp. 33-39
    • Agarwal, S.1    Manchanda, P.2    Vogelbaum, M.A.3    Ohlfest, J.R.4    Elmquist, W.F.5
  • 167
    • 84860193946 scopus 로고    scopus 로고
    • Intranasal delivery of biologics to the central nervous system
    • PID: 22119441, COI: 1:CAS:528:DC%2BC38XlvFyrsL4%3D
    • Lochhead JJ, Thorne RG. Intranasal delivery of biologics to the central nervous system. Adv Drug Deliv Rev 2012;64:614–628.
    • (2012) Adv Drug Deliv Rev , vol.64 , pp. 614-628
    • Lochhead, J.J.1    Thorne, R.G.2
  • 168
    • 0037197831 scopus 로고    scopus 로고
    • Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood-brain barrier
    • PID: 11983877, COI: 1:CAS:528:DC%2BD38XjslWgtro%3D
    • Sly WS, Vogler C. Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood-brain barrier. Proc Natl Acad Sci USA 2002;99:5760–5762.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 5760-5762
    • Sly, W.S.1    Vogler, C.2
  • 169
    • 84899413012 scopus 로고    scopus 로고
    • Glucocorticoids and endothelial cell barrier function
    • PID: 24352805, COI: 1:CAS:528:DC%2BC3sXhvFGhsrvM
    • Salvador E, Shityakov S, Forster C. Glucocorticoids and endothelial cell barrier function. Cell Tissue Res 2014;355:597–605.
    • (2014) Cell Tissue Res , vol.355 , pp. 597-605
    • Salvador, E.1    Shityakov, S.2    Forster, C.3
  • 170
    • 0017383461 scopus 로고
    • Penetration of delta-9-tetrahydrocannabinol and 11-OH-delta-9-tetrahydrocannabinol through the blood-brain barrier
    • COI: 1:CAS:528:DyaE2sXlvFemsrs%3D
    • Schou J, Prockop LD, Dahlstrom G, Rohde C. Penetration of delta-9-tetrahydrocannabinol and 11-OH-delta-9-tetrahydrocannabinol through the blood-brain barrier. Acta Pharmacol Toxicol (Copenh) 1977;41:33–38.
    • (1977) Acta Pharmacol Toxicol (Copenh) , vol.41 , pp. 33-38
    • Schou, J.1    Prockop, L.D.2    Dahlstrom, G.3    Rohde, C.4
  • 171
    • 0035037852 scopus 로고    scopus 로고
    • Transport of human immunodeficiency virus type 1 pseudoviruses across the blood-brain barrier: role of envelope proteins and adsorptive endocytosis
    • PID: 11312339, COI: 1:STN:280:DC%2BD3M3it1ehsA%3D%3D
    • Banks WA, Freed EO, Wolf KM, et al. Transport of human immunodeficiency virus type 1 pseudoviruses across the blood-brain barrier: role of envelope proteins and adsorptive endocytosis. J Virol 2001;75:4681–4691.
    • (2001) J Virol , vol.75 , pp. 4681-4691
    • Banks, W.A.1    Freed, E.O.2    Wolf, K.M.3
  • 172
    • 4344708057 scopus 로고    scopus 로고
    • Binding, internalization, and membrane incorporation of human immunodeficiency virus-1 at the blood-brain barrier is differentially regulated
    • PID: 15450361, COI: 1:CAS:528:DC%2BD2cXmvFels7k%3D
    • Banks WA, Robinson SM, Wolf KM, Bess JW Jr, Arthur LO. Binding, internalization, and membrane incorporation of human immunodeficiency virus-1 at the blood-brain barrier is differentially regulated. Neuroscience 2004;128:143–153.
    • (2004) Neuroscience , vol.128 , pp. 143-153
    • Banks, W.A.1    Robinson, S.M.2    Wolf, K.M.3    Bess, J.W.4    Arthur, L.O.5
  • 173
    • 84866940778 scopus 로고    scopus 로고
    • Transcellular targeting of fiber- and hexon-modified adenovirus vectors across the brain microvascular endothelial cells in vitro
    • PID: 23029348, COI: 1:CAS:528:DC%2BC38XhsV2jtbvJ
    • Laakkonen JP, Engler T, Romero IA, et al. Transcellular targeting of fiber- and hexon-modified adenovirus vectors across the brain microvascular endothelial cells in vitro. PLoS One 2012;7:e45977.
    • (2012) PLoS One , vol.7 , pp. e45977
    • Laakkonen, J.P.1    Engler, T.2    Romero, I.A.3
  • 174
    • 84867002978 scopus 로고    scopus 로고
    • Regulation of Mct1 by cAMP-dependent internalization in rat brain endothelial cells
    • PID: 22925948, COI: 1:CAS:528:DC%2BC38Xhtl2kurnL
    • Smith JP, Uhernik AL, Li L, Liu Z, Drewes LR. Regulation of Mct1 by cAMP-dependent internalization in rat brain endothelial cells. Brain Res 2012;1480:1–11.
    • (2012) Brain Res , vol.1480 , pp. 1-11
    • Smith, J.P.1    Uhernik, A.L.2    Li, L.3    Liu, Z.4    Drewes, L.R.5
  • 175
    • 84865735385 scopus 로고    scopus 로고
    • The Trojan horse liposome technology for nonviral gene transfer across the blood-brain barrier
    • PID: 22175028
    • Boado RJ, Pardridge WM. The Trojan horse liposome technology for nonviral gene transfer across the blood-brain barrier. J Drug Deliv 2011;2011:296151.
    • (2011) J Drug Deliv , vol.2011 , pp. 296151
    • Boado, R.J.1    Pardridge, W.M.2
  • 176
    • 79955467779 scopus 로고    scopus 로고
    • Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors
    • PID: 21397590, COI: 1:CAS:528:DC%2BC3MXltFaisbc%3D
    • Miyake N, Miyake K, Yamamoto M, Hirai Y, Shimada T. Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors. Brain Res 2011;1389:19–26.
    • (2011) Brain Res , vol.1389 , pp. 19-26
    • Miyake, N.1    Miyake, K.2    Yamamoto, M.3    Hirai, Y.4    Shimada, T.5
  • 177
    • 55949130305 scopus 로고    scopus 로고
    • Diffusion in brain extracellular space
    • PID: 18923183, COI: 1:CAS:528:DC%2BD1cXhtlGgtbnI
    • Sykova E, Nicholson C. Diffusion in brain extracellular space. Physiol Rev 2008;88:1277–1340.
    • (2008) Physiol Rev , vol.88 , pp. 1277-1340
    • Sykova, E.1    Nicholson, C.2
  • 178
    • 84864868331 scopus 로고    scopus 로고
    • Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors
    • PID: 21956692, COI: 1:CAS:528:DC%2BC3MXht1CrtrbK
    • Varadi K, Michelfelder S, Korff T, et al. Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors. Gene Ther 2012;19:800–809.
    • (2012) Gene Ther , vol.19 , pp. 800-809
    • Varadi, K.1    Michelfelder, S.2    Korff, T.3
  • 179
    • 79953677173 scopus 로고    scopus 로고
    • The next step in gene delivery: molecular engineering of adeno-associated virus serotypes
    • PID: 21029739, COI: 1:CAS:528:DC%2BC3MXksFKht7g%3D
    • Wang J, Faust SM, Rabinowitz JE. The next step in gene delivery: molecular engineering of adeno-associated virus serotypes. J Mol Cell Cardiol 2011;50:793–802.
    • (2011) J Mol Cell Cardiol , vol.50 , pp. 793-802
    • Wang, J.1    Faust, S.M.2    Rabinowitz, J.E.3
  • 180
    • 77956291663 scopus 로고    scopus 로고
    • Pardridge WM. Preparation of Trojan horse liposomes (THLs) for gene transfer across the blood-brain barrier. Cold Spring Harb Protoc 2010;2010:pdb prot5407.
    • Pardridge WM. Preparation of Trojan horse liposomes (THLs) for gene transfer across the blood-brain barrier. Cold Spring Harb Protoc 2010;2010:pdb prot5407.
  • 181
    • 33846806039 scopus 로고    scopus 로고
    • Translational research models and novel adjunctive therapies for neuroAIDS
    • PID: 18040821
    • Zink MC. Translational research models and novel adjunctive therapies for neuroAIDS. J Neuroimmune Pharmacol 2007;2:14–19.
    • (2007) J Neuroimmune Pharmacol , vol.2 , pp. 14-19
    • Zink, M.C.1
  • 182
    • 0028826147 scopus 로고
    • Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier disruption
    • PID: 7568227, COI: 1:CAS:528:DyaK2MXoslCmtrc%3D
    • Nilaver G, Muldoon LL, Kroll RA, et al. Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier disruption. Proc Natl Acad Sci USA 1995;92:9829–9833.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 9829-9833
    • Nilaver, G.1    Muldoon, L.L.2    Kroll, R.A.3
  • 183
    • 0029898457 scopus 로고    scopus 로고
    • Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells
    • PID: 8597952, COI: 1:CAS:528:DyaK28XitVKmtrw%3D
    • Lacorazza HD, Flax JD, Snyder EY, Jendoubi M. Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells. Nat Med 1996;2:424–429.
    • (1996) Nat Med , vol.2 , pp. 424-429
    • Lacorazza, H.D.1    Flax, J.D.2    Snyder, E.Y.3    Jendoubi, M.4
  • 184
    • 84869002324 scopus 로고    scopus 로고
    • Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound
    • PID: 22838844, COI: 1:CAS:528:DC%2BC38Xhs1Giu7%2FN
    • Thevenot E, Jordao JF, O’Reilly MA, et al. Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound. Hum Gene Ther 2012;23:1144–1155.
    • (2012) Hum Gene Ther , vol.23 , pp. 1144-1155
    • Thevenot, E.1    Jordao, J.F.2    O’Reilly, M.A.3
  • 185
    • 84889872057 scopus 로고    scopus 로고
    • Development, maintenance and disruption of the blood-brain barrier
    • PID: 24309662, COI: 1:CAS:528:DC%2BC3sXhvVyisLbJ
    • Obermeier B, Daneman R, Ransohoff RM. Development, maintenance and disruption of the blood-brain barrier. Nat Med 2013;19:1584–1596.
    • (2013) Nat Med , vol.19 , pp. 1584-1596
    • Obermeier, B.1    Daneman, R.2    Ransohoff, R.M.3
  • 186
    • 77953231021 scopus 로고    scopus 로고
    • Hypercapnia is a possible determinant of the function of the blood-cerebrospinal fluid barrier in amyotrophic lateral sclerosis
    • PID: 20333464
    • Sussmuth SD, Sperfeld AD, Ludolph AC, Tumani H. Hypercapnia is a possible determinant of the function of the blood-cerebrospinal fluid barrier in amyotrophic lateral sclerosis. Neurochem Res 2010;35:1071–1074.
    • (2010) Neurochem Res , vol.35 , pp. 1071-1074
    • Sussmuth, S.D.1    Sperfeld, A.D.2    Ludolph, A.C.3    Tumani, H.4
  • 187
    • 79952499042 scopus 로고    scopus 로고
    • Disruption of neurovascular unit prior to motor neuron degeneration in amyotrophic lateral sclerosis
    • PID: 21337372, COI: 1:CAS:528:DC%2BC3MXivFCitbg%3D
    • Miyazaki K, Ohta Y, Nagai M, et al. Disruption of neurovascular unit prior to motor neuron degeneration in amyotrophic lateral sclerosis. J Neurosci Res 2011;89:718–728.
    • (2011) J Neurosci Res , vol.89 , pp. 718-728
    • Miyazaki, K.1    Ohta, Y.2    Nagai, M.3
  • 188
    • 84872363503 scopus 로고    scopus 로고
    • Blood-spinal cord barrier breakdown and pericyte reductions in amyotrophic lateral sclerosis
    • PID: 22941226, COI: 1:CAS:528:DC%2BC3sXktVyqsA%3D%3D
    • Winkler EA, Sengillo JD, Sullivan JS, et al. Blood-spinal cord barrier breakdown and pericyte reductions in amyotrophic lateral sclerosis. Acta Neuropathol 2013;125:111–120.
    • (2013) Acta Neuropathol , vol.125 , pp. 111-120
    • Winkler, E.A.1    Sengillo, J.D.2    Sullivan, J.S.3
  • 189
    • 84896520534 scopus 로고    scopus 로고
    • Blood-spinal cord barrier disruption contributes to early motor-neuron degeneration in ALS-model mice
    • PID: 24591593, COI: 1:CAS:528:DC%2BC2cXjtlyrur8%3D
    • Winkler EA, Sengillo JD, Sagare AP, et al. Blood-spinal cord barrier disruption contributes to early motor-neuron degeneration in ALS-model mice. Proc Natl Acad Sci USA 2014;111:E1035-42.
    • (2014) Proc Natl Acad Sci USA , vol.111 , pp. E1035-E1042
    • Winkler, E.A.1    Sengillo, J.D.2    Sagare, A.P.3
  • 190
    • 34250658083 scopus 로고    scopus 로고
    • Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS
    • PID: 17512910, COI: 1:CAS:528:DC%2BD2sXmvVOit7s%3D
    • Garbuzova-Davis S, Haller E, Saporta S, et al. Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS. Brain Res 2007;1157:126–137.
    • (2007) Brain Res , vol.1157 , pp. 126-137
    • Garbuzova-Davis, S.1    Haller, E.2    Saporta, S.3
  • 191
    • 77951206888 scopus 로고    scopus 로고
    • Blood-brain barrier abnormalities caused by exposure to HIV-1 gp120—protection by gene delivery of antioxidant enzymes
    • PID: 20219678, COI: 1:CAS:528:DC%2BC3cXksFCmtbc%3D
    • Louboutin JP, Reyes BA, Agrawal L, et al. Blood-brain barrier abnormalities caused by exposure to HIV-1 gp120—protection by gene delivery of antioxidant enzymes. Neurobiol Dis 2010;38:313–325.
    • (2010) Neurobiol Dis , vol.38 , pp. 313-325
    • Louboutin, J.P.1    Reyes, B.A.2    Agrawal, L.3
  • 192
    • 18644367631 scopus 로고    scopus 로고
    • Expression polymorphism of the blood-brain barrier component P-glycoprotein (MDR1) in relation to Parkinson’s disease
    • PID: 12360103, COI: 1:CAS:528:DC%2BD38XnsVeqtbo%3D
    • Furuno T, Landi MT, Ceroni M, et al. Expression polymorphism of the blood-brain barrier component P-glycoprotein (MDR1) in relation to Parkinson’s disease. Pharmacogenetics 2002;12:529–534.
    • (2002) Pharmacogenetics , vol.12 , pp. 529-534
    • Furuno, T.1    Landi, M.T.2    Ceroni, M.3
  • 193
    • 46649085260 scopus 로고    scopus 로고
    • Decreased blood-brain barrier P-glycoprotein function in the progression of Parkinson’s disease, PSP and MSA
    • PID: 18265929, COI: 1:CAS:528:DC%2BD1cXotV2mtLY%3D
    • Bartels AL, Willemsen AT, Kortekaas R, et al. Decreased blood-brain barrier P-glycoprotein function in the progression of Parkinson’s disease, PSP and MSA. J Neural Transm 2008;115:1001–1009.
    • (2008) J Neural Transm , vol.115 , pp. 1001-1009
    • Bartels, A.L.1    Willemsen, A.T.2    Kortekaas, R.3
  • 194
    • 1342326087 scopus 로고    scopus 로고
    • Glioblastoma cells release factors that disrupt blood-brain barrier features
    • PID: 14730455
    • Schneider SW, Ludwig T, Tatenhorst L, et al. Glioblastoma cells release factors that disrupt blood-brain barrier features. Acta Neuropathol 2004;107:272–276.
    • (2004) Acta Neuropathol , vol.107 , pp. 272-276
    • Schneider, S.W.1    Ludwig, T.2    Tatenhorst, L.3
  • 195
    • 43249103996 scopus 로고    scopus 로고
    • Endothelial cell barrier impairment induced by glioblastomas and transforming growth factor beta2 involves matrix metalloproteinases and tight junction proteins
    • PID: 18431253, COI: 1:CAS:528:DC%2BD1cXmsFehurw%3D
    • Ishihara, H., H. Kubota, R.L. Lindberg, et al., Endothelial cell barrier impairment induced by glioblastomas and transforming growth factor beta2 involves matrix metalloproteinases and tight junction proteins. J Neuropathol Exp Neurol 2008;67:435–448.
    • (2008) J Neuropathol Exp Neurol , vol.67 , pp. 435-448
    • Ishihara, H.1    Kubota, H.2    Lindberg, R.L.3
  • 196
    • 84869852211 scopus 로고    scopus 로고
    • Cerebrospinal fluid matrix metalloproteinases are elevated in cerebral adrenoleukodystrophy and correlate with MRI severity and neurologic dysfunction
    • PID: 23185624, COI: 1:CAS:528:DC%2BC38XhvVaqtL7I
    • Thibert KA, Raymond GV, Nascene DR, et al. Cerebrospinal fluid matrix metalloproteinases are elevated in cerebral adrenoleukodystrophy and correlate with MRI severity and neurologic dysfunction. PLoS One 2012;7:e50430.
    • (2012) PLoS One , vol.7 , pp. e50430
    • Thibert, K.A.1    Raymond, G.V.2    Nascene, D.R.3
  • 197
    • 70350502352 scopus 로고    scopus 로고
    • Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
    • PID: 19749771, COI: 1:CAS:528:DC%2BD1MXhtFaju77J
    • Chen YH, Chang M, Davidson BL. Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med 2009;15:1215–1218.
    • (2009) Nat Med , vol.15 , pp. 1215-1218
    • Chen, Y.H.1    Chang, M.2    Davidson, B.L.3
  • 198
    • 33745751979 scopus 로고    scopus 로고
    • Apolipoprotein E-derived peptides reduce CNS inflammation: implications for therapy of neurological disease
    • PID: 16866906, COI: 1:STN:280:DC%2BD28vktlejsA%3D%3D
    • Laskowitz DT, Fillit H, Yeung N, Toku K, Vitek MP. Apolipoprotein E-derived peptides reduce CNS inflammation: implications for therapy of neurological disease. Acta Neurol Scand Suppl 2006;185:15–20.
    • (2006) Acta Neurol Scand Suppl , vol.185 , pp. 15-20
    • Laskowitz, D.T.1    Fillit, H.2    Yeung, N.3    Toku, K.4    Vitek, M.P.5
  • 199
    • 45749090515 scopus 로고    scopus 로고
    • Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse
    • PID: 18443601, COI: 1:CAS:528:DC%2BD1cXns1eiu7Y%3D
    • Urayama A, Grubb JH, Sly WS, Banks WA. Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse. Mol Ther 2008;16:1261–1266.
    • (2008) Mol Ther , vol.16 , pp. 1261-1266
    • Urayama, A.1    Grubb, J.H.2    Sly, W.S.3    Banks, W.A.4
  • 200
    • 0036202298 scopus 로고    scopus 로고
    • Critical issues in gene therapy for neurologic disease
    • PID: 11916483, COI: 1:CAS:528:DC%2BD38Xkt1Srsb4%3D
    • Hsich G, Sena-Esteves M, Breakefield XO. Critical issues in gene therapy for neurologic disease. Hum Gene Ther 2002;13:579–604.
    • (2002) Hum Gene Ther , vol.13 , pp. 579-604
    • Hsich, G.1    Sena-Esteves, M.2    Breakefield, X.O.3
  • 201
    • 84895442417 scopus 로고    scopus 로고
    • Parkinson’s disease gene therapy: success by design meets failure by efficacy
    • PID: 24356252, COI: 1:CAS:528:DC%2BC2cXps1Gmtg%3D%3D
    • Bartus RT, Weinberg MS, Samulski RJ. Parkinson’s disease gene therapy: success by design meets failure by efficacy. Mol Ther 2014;22:487–497.
    • (2014) Mol Ther , vol.22 , pp. 487-497
    • Bartus, R.T.1    Weinberg, M.S.2    Samulski, R.J.3
  • 202
    • 33645145491 scopus 로고    scopus 로고
    • Large animal models and gene therapy
    • PID: 16333317, COI: 1:CAS:528:DC%2BD28Xhs1eqsbk%3D
    • Casal M, Haskins M. Large animal models and gene therapy. Eur J Hum Genet 2006;14:266–272
    • (2006) Eur J Hum Genet , vol.14 , pp. 266-272
    • Casal, M.1    Haskins, M.2
  • 203
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • PID: 16474400, COI: 1:CAS:528:DC%2BD28XitVGnt78%3D
    • Manno CS, Pierce GF, Arruda VR et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342–347.
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 204
    • 84894242894 scopus 로고    scopus 로고
    • Selection and evaluation of clinically relevant AAV variants in a xenograft liver model
    • PID: 24390344, COI: 1:CAS:528:DC%2BC2cXivVenurs%3D
    • Lisowski L, Dane AP, Chu K, et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature 2014;506:382–386.
    • (2014) Nature , vol.506 , pp. 382-386
    • Lisowski, L.1    Dane, A.P.2    Chu, K.3
  • 205
    • 0030866841 scopus 로고    scopus 로고
    • Benefits and risks of hosting animal cells in the human brain
    • PID: 9288721, COI: 1:CAS:528:DyaK2sXlvVynurs%3D
    • Isacson O, Breakefield XO. Benefits and risks of hosting animal cells in the human brain. Nat Med 1997;3:964–969.
    • (1997) Nat Med , vol.3 , pp. 964-969
    • Isacson, O.1    Breakefield, X.O.2
  • 206
    • 84878581647 scopus 로고    scopus 로고
    • Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2
    • PID: 23474247
    • Testa F, Maguire AM, Rossi S, et al. Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology 2013;120:1283–1291.
    • (2013) Ophthalmology , vol.120 , pp. 1283-1291
    • Testa, F.1    Maguire, A.M.2    Rossi, S.3
  • 207
    • 84908200336 scopus 로고    scopus 로고
    • fMRI of retina-originated phosphenes experienced by patients with leber congenital amaurosis
    • PID: 24465873
    • Ashtari M, Cyckowski L, Yazdi A, et al. fMRI of retina-originated phosphenes experienced by patients with leber congenital amaurosis. PLoS One 2014;9:e86068.
    • (2014) PLoS One , vol.9 , pp. e86068
    • Ashtari, M.1    Cyckowski, L.2    Yazdi, A.3
  • 208
    • 84864297772 scopus 로고    scopus 로고
    • Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy
    • PID: 22841313, COI: 1:CAS:528:DC%2BC38XhtV2ms7vP
    • Akil O, Seal RP, Burke K, et al. Restoration of hearing in the VGLUT3 knockout mouse using virally mediated gene therapy. Neuron 2012;75:283–293.
    • (2012) Neuron , vol.75 , pp. 283-293
    • Akil, O.1    Seal, R.P.2    Burke, K.3
  • 209
    • 84866887475 scopus 로고    scopus 로고
    • Gene therapy for deaf mice goes viral
    • PID: 23023054, COI: 1:CAS:528:DC%2BC38XhsVaqt73K
    • Holt JR, Vandenberghe LH. Gene therapy for deaf mice goes viral. Mol Ther 2012;20 1836–1837.
    • (2012) Mol Ther , vol.20 , pp. 1836-1837
    • Holt, J.R.1    Vandenberghe, L.H.2
  • 210
    • 79952976864 scopus 로고    scopus 로고
    • Convection-enhanced delivery to achieve widespread distribution of viral vectors: predicting clinical implementation
    • PID: 21154156, COI: 1:CAS:528:DC%2BC3MXhvVelu7w%3D
    • Debinski W, Tatter SB. Convection-enhanced delivery to achieve widespread distribution of viral vectors: predicting clinical implementation. Curr Opin Mol Ther 2010;12:647–653.
    • (2010) Curr Opin Mol Ther , vol.12 , pp. 647-653
    • Debinski, W.1    Tatter, S.B.2
  • 211
    • 84892474913 scopus 로고    scopus 로고
    • Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis
    • PID: 24108104, COI: 1:CAS:528:DC%2BC2cXlvFehtA%3D%3D
    • Wang H, Yang B, Qiu L et al. Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis. Hum Mol Genet 2014;23:668–681.
    • (2014) Hum Mol Genet , vol.23 , pp. 668-681
    • Wang, H.1    Yang, B.2    Qiu, L.3
  • 212
    • 84857579405 scopus 로고    scopus 로고
    • Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy
    • PID: 22365775, COI: 1:CAS:528:DC%2BC38XlvFKjsrY%3D
    • Cartier N, Hacein-Bey-Abina S, Bartholomae CC, et al. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy. Methods Enzymol 2012;507:187–198.
    • (2012) Methods Enzymol , vol.507 , pp. 187-198
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3
  • 213
    • 55949109698 scopus 로고    scopus 로고
    • Seven-Tesla proton magnetic resonance spectroscopic imaging in adult X-linked adrenoleukodystrophy
    • PID: 19001168
    • Ratai E, Kok T, Wiggins C, et al. Seven-Tesla proton magnetic resonance spectroscopic imaging in adult X-linked adrenoleukodystrophy. Arch Neurol 2008;65:1488–1494.
    • (2008) Arch Neurol , vol.65 , pp. 1488-1494
    • Ratai, E.1    Kok, T.2    Wiggins, C.3
  • 214
    • 84882384439 scopus 로고    scopus 로고
    • Toxicology and biodistribution studies for MGH2.1, an oncolytic virus that expresses two prodrug-activating genes, in combination with prodrugs
    • Kasai K, Nakashima H, Liu F, et al. Toxicology and biodistribution studies for MGH2.1, an oncolytic virus that expresses two prodrug-activating genes, in combination with prodrugs. Mol Ther Nucleic Acids 2013;6:e113.
    • (2013) Mol Ther Nucleic Acids , vol.6 , pp. e113
    • Kasai, K.1    Nakashima, H.2    Liu, F.3
  • 215
    • 0034619328 scopus 로고    scopus 로고
    • Neural stem cells display extensive tropism for pathology in adult brain: evidence from intracranial gliomas
    • PID: 11070094, COI: 1:CAS:528:DC%2BD3cXotFyksrc%3D
    • Aboody KS, Brown A, Rainov NG, et al. Neural stem cells display extensive tropism for pathology in adult brain: evidence from intracranial gliomas. Proc Natl Acad Sci USA 2000;97:12846–12851.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 12846-12851
    • Aboody, K.S.1    Brown, A.2    Rainov, N.G.3
  • 216
    • 84888395554 scopus 로고    scopus 로고
    • Neural stem cell-mediated delivery of irinotecan-activating carboxylesterases to glioma: implications for clinical use
    • PID: 24167321, COI: 1:CAS:528:DC%2BC2cXitF2msA%3D%3D
    • Metz MZ, Gutova M, Lacey SF, et al. Neural stem cell-mediated delivery of irinotecan-activating carboxylesterases to glioma: implications for clinical use. Stem Cells Transl Med 2013;2:983–992.
    • (2013) Stem Cells Transl Med , vol.2 , pp. 983-992
    • Metz, M.Z.1    Gutova, M.2    Lacey, S.F.3
  • 217
    • 84898604804 scopus 로고    scopus 로고
    • Development of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles
    • Rodríguez-Gascón A, Del Pozo-Rodríguez A, Solinís MA. Development of nucleic acid vaccines: use of self-amplifying RNA in lipid nanoparticles. Int J Nanomed 2014;9:1833–1843.
    • (2014) Int J Nanomed , vol.9 , pp. 1833-1843
    • Rodríguez-Gascón, A.1    Del Pozo-Rodríguez, A.2    Solinís, M.A.3
  • 218
    • 84871923988 scopus 로고    scopus 로고
    • Genetically engineered microvesicles carrying suicide mRNA/protein inhibit schwannoma tumor growth
    • PID: 22910294, COI: 1:CAS:528:DC%2BC38Xht1akt7%2FI
    • Mizrak A, Bolukbasi MF, Ozdener GB, et al. Genetically engineered microvesicles carrying suicide mRNA/protein inhibit schwannoma tumor growth. Mol Ther 2013;21:101–108.
    • (2013) Mol Ther , vol.21 , pp. 101-108
    • Mizrak, A.1    Bolukbasi, M.F.2    Ozdener, G.B.3
  • 219
    • 84920767728 scopus 로고    scopus 로고
    • György B, Marcus ME, Breakefield XO, Leonard JN. Therapeutic applications of extracellular vesicles—clinical promise and open questions. Ann Rev Pharm Tox 2015:55; [Epub ahead of print].
    • György B, Marcus ME, Breakefield XO, Leonard JN. Therapeutic applications of extracellular vesicles—clinical promise and open questions. Ann Rev Pharm Tox 2015:55; [Epub ahead of print].
  • 220
    • 67650828361 scopus 로고    scopus 로고
    • Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA
    • PID: 19608921, COI: 1:CAS:528:DC%2BD1MXos1Sqt7c%3D
    • Wheeler TM, Sobczak K, Lueck JD, et al. Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA. Science 2009;325:336–339.
    • (2009) Science , vol.325 , pp. 336-339
    • Wheeler, T.M.1    Sobczak, K.2    Lueck, J.D.3
  • 221
    • 84864439453 scopus 로고    scopus 로고
    • Targeting nuclear RNA for in vivo correction of myotonic dystrophy
    • PID: 22859208, COI: 1:CAS:528:DC%2BC38XhtFChu7fK
    • Wheeler TM, Leger AJ, Pandey SK, et al. Targeting nuclear RNA for in vivo correction of myotonic dystrophy. Nature 2012;488:111–115.
    • (2012) Nature , vol.488 , pp. 111-115
    • Wheeler, T.M.1    Leger, A.J.2    Pandey, S.K.3
  • 222
    • 84885808774 scopus 로고    scopus 로고
    • RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention
    • PID: 24139042, COI: 1:CAS:528:DC%2BC3sXhs1CrurfK
    • Donnelly CJ, Zhang PW, Pham JT, et al. RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention. Neuron 2013;80:415–428.
    • (2013) Neuron , vol.80 , pp. 415-428
    • Donnelly, C.J.1    Zhang, P.W.2    Pham, J.T.3
  • 223
    • 84886389563 scopus 로고    scopus 로고
    • Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion
    • PID: 24154603
    • Sareen D, O’Rourke JG, Meera P, et al. Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion. Sci Transl Med 2013;5:208ra149.
    • (2013) Sci Transl Med , vol.5 , pp. 208ra149
    • Sareen, D.1    O’Rourke, J.G.2    Meera, P.3
  • 224
    • 84888098632 scopus 로고    scopus 로고
    • Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration
    • PID: 24170860, COI: 1:CAS:528:DC%2BC3sXhvFCisL3N
    • Lagier-Tourenne C, Baughn M, Rigo F, et al. Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration. Proc Natl Acad Sci USA 2013;110:E4530–4539.
    • (2013) Proc Natl Acad Sci USA , vol.110 , pp. E4530-E4539
    • Lagier-Tourenne, C.1    Baughn, M.2    Rigo, F.3
  • 225
    • 84865688581 scopus 로고    scopus 로고
    • Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression
    • PID: 22939619, COI: 1:CAS:528:DC%2BC38XhtlSnsrvJ
    • Yu D, Pendergraff H, Liu J, et al. Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 2012;150:895–908.
    • (2012) Cell , vol.150 , pp. 895-908
    • Yu, D.1    Pendergraff, H.2    Liu, J.3
  • 226
    • 84862663712 scopus 로고    scopus 로고
    • Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis
    • PID: 22726834, COI: 1:CAS:528:DC%2BC38XptVKgtbs%3D
    • Kordasiewicz HB, Stanek LM, Wancewicz EV, et al. Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis. Neuron 2012;74:1031–1044.
    • (2012) Neuron , vol.74 , pp. 1031-1044
    • Kordasiewicz, H.B.1    Stanek, L.M.2    Wancewicz, E.V.3
  • 227
    • 84899918530 scopus 로고    scopus 로고
    • Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy
    • PID: 24746732, COI: 1:CAS:528:DC%2BC2cXmtlCmtrc%3D
    • Lieberman AP, Yu Z, Murray S, et al. Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. Cell Rep 2014;7:774–784.
    • (2014) Cell Rep , vol.7 , pp. 774-784
    • Lieberman, A.P.1    Yu, Z.2    Murray, S.3
  • 228
    • 84896352928 scopus 로고    scopus 로고
    • Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy δ7 mouse model phenotype
    • PID: 24636820, COI: 1:CAS:528:DC%2BC2cXkslSqur0%3D
    • Nizzardo M, Simone C, Salani S, et al. Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy δ7 mouse model phenotype. Clin Ther 2014;36:340–356.
    • (2014) Clin Ther , vol.36 , pp. 340-356
    • Nizzardo, M.1    Simone, C.2    Salani, S.3
  • 230
    • 84900835509 scopus 로고    scopus 로고
    • New developments in exon skipping and splice modulation therapies for neuromuscular diseases
    • PID: 24620745, COI: 1:CAS:528:DC%2BC2cXot1yntL4%3D
    • Touznik A, Lee JJ, Yokota T. New developments in exon skipping and splice modulation therapies for neuromuscular diseases. Expert Opin Biol Ther 2014;14:809–819.
    • (2014) Expert Opin Biol Ther , vol.14 , pp. 809-819
    • Touznik, A.1    Lee, J.J.2    Yokota, T.3
  • 231
    • 84888321603 scopus 로고    scopus 로고
    • Lipid-polymer hybrid nanoparticles as a new generation therapeutic delivery platform: a review
    • PID: 23872180, COI: 1:CAS:528:DC%2BC3sXhtlWhtLrI
    • Hadinoto K, Sundaresan A, Cheow WS. Lipid-polymer hybrid nanoparticles as a new generation therapeutic delivery platform: a review. Eur J Pharm Biopharm 2013;85:427–443.
    • (2013) Eur J Pharm Biopharm , vol.85 , pp. 427-443
    • Hadinoto, K.1    Sundaresan, A.2    Cheow, W.S.3
  • 232
    • 84876466100 scopus 로고    scopus 로고
    • An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
    • PID: 23541756, COI: 1:CAS:528:DC%2BC3sXltVynsrY%3D
    • Miller TM, Pestronk A, David W, et al. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol 2013;12:435–442.
    • (2013) Lancet Neurol , vol.12 , pp. 435-442
    • Miller, T.M.1    Pestronk, A.2    David, W.3
  • 233
    • 79960981599 scopus 로고    scopus 로고
    • Targeting RNA to treat neuromuscular disease
    • PID: 21804598, COI: 1:CAS:528:DC%2BC3MXpsFKquro%3D
    • Muntoni F, Wood MJ. Targeting RNA to treat neuromuscular disease. Nat Rev Drug Discov 2011;10:621–637.
    • (2011) Nat Rev Drug Discov , vol.10 , pp. 621-637
    • Muntoni, F.1    Wood, M.J.2
  • 234
    • 84877872340 scopus 로고    scopus 로고
    • Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy
    • PID: 23521559, COI: 1:CAS:528:DC%2BC3sXns1ahs78%3D
    • Koo T, Wood MJ. Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy. Hum Gene Ther 2013;24:479–488.
    • (2013) Hum Gene Ther , vol.24 , pp. 479-488
    • Koo, T.1    Wood, M.J.2
  • 235
    • 82955199935 scopus 로고    scopus 로고
    • Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington’s disease
    • PID: 22031240, COI: 1:CAS:528:DC%2BC3MXhtlOnur3I
    • McBride JL, Pitzer MR, Boudreau RL, et al. Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington’s disease. Mol Ther 2011;19:2152–2162.
    • (2011) Mol Ther , vol.19 , pp. 2152-2162
    • McBride, J.L.1    Pitzer, M.R.2    Boudreau, R.L.3
  • 236
    • 84895468010 scopus 로고    scopus 로고
    • Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy
    • PID: 24419082, COI: 1:CAS:528:DC%2BC2cXmtlWksg%3D%3D
    • Keiser MS, Boudreau RL, Davidson BL. Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy. Mol Ther 2014;22:588–595.
    • (2014) Mol Ther , vol.22 , pp. 588-595
    • Keiser, M.S.1    Boudreau, R.L.2    Davidson, B.L.3
  • 237
    • 84883118140 scopus 로고    scopus 로고
    • Safety and efficacy of RNAi therapy for transthyretin amyloidosis
    • PID: 23984729, COI: 1:CAS:528:DC%2BC3sXhtlyis7fK
    • Coelho T, Adams D, Silva A, et al. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med 2013;369:819–829.
    • (2013) N Engl J Med , vol.369 , pp. 819-829
    • Coelho, T.1    Adams, D.2    Silva, A.3
  • 238
    • 84897458124 scopus 로고    scopus 로고
    • Targeted genome editing tools for disease modeling and gene therapy
    • PID: 24665839, COI: 1:CAS:528:DC%2BC2cXlt1eru74%3D
    • Cai M, Yang Y. Targeted genome editing tools for disease modeling and gene therapy. Curr Gene Ther 2014;14:2–9.
    • (2014) Curr Gene Ther , vol.14 , pp. 2-9
    • Cai, M.1    Yang, Y.2
  • 239
    • 84871519181 scopus 로고    scopus 로고
    • TALENs: a widely applicable technology for targeted genome editing
    • PID: 23169466, COI: 1:CAS:528:DC%2BC38Xhs12ks7rJ
    • Joung JK, Sander JD TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol 2013;14:49–55.
    • (2013) Nat Rev Mol Cell Biol , vol.14 , pp. 49-55
    • Joung, J.K.1    Sander, J.D.2
  • 240
    • 84884288934 scopus 로고    scopus 로고
    • Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
    • PID: 23992846, COI: 1:CAS:528:DC%2BC3sXhtlGrur3M
    • Ran FA, Hsu PD, Lin CY, et al. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 2013;154:1380–1389.
    • (2013) Cell , vol.154 , pp. 1380-1389
    • Ran, F.A.1    Hsu, P.D.2    Lin, C.Y.3
  • 241
    • 84879264708 scopus 로고    scopus 로고
    • ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
    • Gaj T, Gersbach CA, Barbas CF. ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends Biotechnol 2014;31:397–405.
    • (2014) Trends Biotechnol , vol.31 , pp. 397-405
    • Gaj, T.1    Gersbach, C.A.2    Barbas, C.F.3
  • 242
    • 84887506882 scopus 로고    scopus 로고
    • LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson’s disease patients: reversal by gene correction
    • PID: 24148854, COI: 1:CAS:528:DC%2BC2cXjt1Ojsw%3D%3D
    • Sanders LH, Laganière J, Cooper O, et al. LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson’s disease patients: reversal by gene correction. Neurobiol Dis 2014;62:381–386.
    • (2014) Neurobiol Dis , vol.62 , pp. 381-386
    • Sanders, L.H.1    Laganière, J.2    Cooper, O.3
  • 243
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • PID: 23287718, COI: 1:CAS:528:DC%2BC3sXit1ygtb8%3D
    • Cong L, Ran FA, Cox D, et al. Multiplex genome engineering using CRISPR/Cas systems. Science 2013;339:819–823.
    • (2013) Science , vol.339 , pp. 819-823
    • Cong, L.1    Ran, F.A.2    Cox, D.3
  • 244
    • 84887772816 scopus 로고    scopus 로고
    • Newer gene editing technologies toward HIV gene therapy
    • PID: 24284874, COI: 1:CAS:528:DC%2BC2cXmsVyjsLY%3D
    • Manjunath N, Yi G, Dang Y, Shankar P. Newer gene editing technologies toward HIV gene therapy. Viruses 2013;5:2748–2766.
    • (2013) Viruses , vol.5 , pp. 2748-2766
    • Manjunath, N.1    Yi, G.2    Dang, Y.3    Shankar, P.4
  • 245
    • 84902095353 scopus 로고    scopus 로고
    • Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
    • PID: 24681508, COI: 1:CAS:528:DC%2BC2cXltFKmt7Y%3D
    • Yin H, Xue W, Chen S et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 2014;32:551–553.
    • (2014) Nat Biotechnol , vol.32 , pp. 551-553
    • Yin, H.1    Xue, W.2    Chen, S.3
  • 246
    • 84890048526 scopus 로고    scopus 로고
    • Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins
    • PID: 24108092, COI: 1:CAS:528:DC%2BC3sXhsFymsLjP
    • Maeder ML, Angstman JF, Richardson ME, et al. Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins. Nat Biotechnol 2013;31:1137–1142.
    • (2013) Nat Biotechnol , vol.31 , pp. 1137-1142
    • Maeder, M.L.1    Angstman, J.F.2    Richardson, M.E.3
  • 247
    • 84886084801 scopus 로고    scopus 로고
    • Locus-specific editing of histone modifications at endogenous enhancers
    • PID: 24013198, COI: 1:CAS:528:DC%2BC3sXhtl2kt7vO
    • Mendenhall EM, Williamson KE, Reyon D, et al. Locus-specific editing of histone modifications at endogenous enhancers. Nat Biotechnol 2013;31:1133–1136.
    • (2013) Nat Biotechnol , vol.31 , pp. 1133-1136
    • Mendenhall, E.M.1    Williamson, K.E.2    Reyon, D.3
  • 248
    • 85027921807 scopus 로고    scopus 로고
    • Rafii MS, Baumann TL, Bakay RA, et al. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease. Alzheimers Dement 2014 Jan 7 [Epub ahead of print].
    • Rafii MS, Baumann TL, Bakay RA, et al. A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer’s disease. Alzheimers Dement 2014 Jan 7 [Epub ahead of print].
  • 249
    • 84879086501 scopus 로고    scopus 로고
    • Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
    • PID: 23576625, COI: 1:CAS:528:DC%2BC3sXmvFSrtLw%3D
    • Bartus RT, Baumann TL, Siffert J, et al. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 2013;80:1698–1701.
    • (2013) Neurology , vol.80 , pp. 1698-1701
    • Bartus, R.T.1    Baumann, T.L.2    Siffert, J.3
  • 250
    • 0033837214 scopus 로고    scopus 로고
    • Glial cell line-derived neurotrophic factor (GDNF) as a defensive molecule for neurodegenerative disease: a tribute to the studies of antonia vernadakis on neuronal-glial interactions
    • PID: 10978846, COI: 1:CAS:528:DC%2BD3cXmtF2hsb0%3D
    • Bohn MC, Kozlowski DA, Connor B. Glial cell line-derived neurotrophic factor (GDNF) as a defensive molecule for neurodegenerative disease: a tribute to the studies of antonia vernadakis on neuronal-glial interactions. Int J Dev Neurosci 2000;18:679–684.
    • (2000) Int J Dev Neurosci , vol.18 , pp. 679-684
    • Bohn, M.C.1    Kozlowski, D.A.2    Connor, B.3
  • 251
    • 58149242839 scopus 로고    scopus 로고
    • Subcutaneous IGF-1 is not beneficial in 2-year ALS trial
    • PID: 19029516, COI: 1:CAS:528:DC%2BD1cXhtlylurjK
    • Sorenson EJ, Windbank AJ, Mandrekar JN, et al. Subcutaneous IGF-1 is not beneficial in 2-year ALS trial. Neurology 2008;71:1770–1775.
    • (2008) Neurology , vol.71 , pp. 1770-1775
    • Sorenson, E.J.1    Windbank, A.J.2    Mandrekar, J.N.3
  • 252
    • 84905455009 scopus 로고    scopus 로고
    • Network modulation following sham surgery in Parkinson’s
    • PID: 25036712, COI: 1:CAS:528:DC%2BC2cXhtlGkt7rF
    • Ko JH, Feigin A, Mattis PJ, et al. Network modulation following sham surgery in Parkinson’s. J Clin Invest 2014;124:3656–3666.
    • (2014) J Clin Invest , vol.124 , pp. 3656-3666
    • Ko, J.H.1    Feigin, A.2    Mattis, P.J.3
  • 253
    • 84862140284 scopus 로고    scopus 로고
    • Sustained induction of neuronal addition to the adult rat neostriatum by AAV4-delivered noggin and BDNF
    • PID: 21918547, COI: 1:CAS:528:DC%2BC3MXhtFOls7zL
    • Benraiss A, Bruel-Jungerman E, Lu G, et al. Sustained induction of neuronal addition to the adult rat neostriatum by AAV4-delivered noggin and BDNF. Gene Ther 2012;19:483–493.
    • (2012) Gene Ther , vol.19 , pp. 483-493
    • Benraiss, A.1    Bruel-Jungerman, E.2    Lu, G.3
  • 254
    • 84890365334 scopus 로고    scopus 로고
    • Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain
    • PID: 24259049
    • Hudry E, Dashkoff J, Roe AD, et al. Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain. Sci Transl Med 2013;5:212ra161.
    • (2013) Sci Transl Med , vol.5 , pp. 212ra161
    • Hudry, E.1    Dashkoff, J.2    Roe, A.D.3
  • 255
    • 84895904922 scopus 로고    scopus 로고
    • Treatment of type 1 myotonic dystrophy by engineering site-specific RNA endonucleases that target (CUG)(n) repeats
    • PID: 24196578, COI: 1:CAS:528:DC%2BC3sXhvVKgsbrJ
    • Zhang W, Wang Y, Dong S, et al. Treatment of type 1 myotonic dystrophy by engineering site-specific RNA endonucleases that target (CUG)(n) repeats. Mol Ther 2014;22:312–320.
    • (2014) Mol Ther , vol.22 , pp. 312-320
    • Zhang, W.1    Wang, Y.2    Dong, S.3
  • 256
    • 84856431819 scopus 로고    scopus 로고
    • RNA therapeutics: beyond RNA interference and antisense oligonucleotides
    • PID: 22262036, COI: 1:CAS:528:DC%2BC38XhtVGqt7k%3D
    • Kole R, Krainer AR, Altman S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 2012;11:125–140.
    • (2012) Nat Rev Drug Discov , vol.11 , pp. 125-140
    • Kole, R.1    Krainer, A.R.2    Altman, S.3
  • 257
    • 84883491304 scopus 로고    scopus 로고
    • Metabolic network as a progression biomarker of premanifest Huntington’s disease
    • PID: 23985564, COI: 1:CAS:528:DC%2BC3sXhsVemsrzK
    • Tang CC, Feigin A, Ma Y, et al. Metabolic network as a progression biomarker of premanifest Huntington’s disease. J Clin Invest 2013;123:4076–4088.
    • (2013) J Clin Invest , vol.123 , pp. 4076-4088
    • Tang, C.C.1    Feigin, A.2    Ma, Y.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.