메뉴 건너뛰기




Volumn 1382, Issue , 2016, Pages 133-149

Controlling AAV tropism in the nervous system with natural and engineered capsids

Author keywords

AAV; Adeno associated virus; Capsid; Gene delivery; Gene therapy; Serotype; Vector; Virus

Indexed keywords

PARVOVIRUS VECTOR;

EID: 84948844867     PISSN: 10643745     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-4939-3271-9_10     Document Type: Chapter
Times cited : (109)

References (131)
  • 1
    • 0021690155 scopus 로고
    • Use of adeno-associated virus as a mammalian DNA cloning vector: Transduction of neomycin resistance into mammalian tissue culture cells
    • Hermonat PL, Muzyczka N (1984) Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells. Proc Natl Acad Sci U S A 81: 6466–6470
    • (1984) Proc Natl Acad Sci U S A , vol.81 , pp. 6466-6470
    • Hermonat, P.L.1    Muzyczka, N.2
  • 2
    • 0010390169 scopus 로고
    • Cloning of adeno-associated virus into pBR322: Rescue of intact virus from the recombinant plasmid in human cells
    • Samulski RJ, Berns KI, Tan M, Muzyczka N (1982) Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc Natl Acad Sci U S A 79:2077–2081
    • (1982) Proc Natl Acad Sci U S A , vol.79 , pp. 2077-2081
    • Samulski, R.J.1    Berns, K.I.2    Tan, M.3    Muzyczka, N.4
  • 3
    • 0023216839 scopus 로고
    • A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication
    • Samulski RJ, Chang LS, Shenk T (1987) A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol 61:3096–3101
    • (1987) J Virol , vol.61 , pp. 3096-3101
    • Samulski, R.J.1    Chang, L.S.2    Shenk, T.3
  • 4
    • 0021716589 scopus 로고
    • A human parvovirus, adenoassociated virus, as a eucaryotic vector: Transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase
    • Tratschin JD, West MH, Sandbank T, Carter BJ (1984) A human parvovirus, adenoassociated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol 4:2072–2081
    • (1984) Mol Cell Biol , vol.4 , pp. 2072-2081
    • Tratschin, J.D.1    West, M.H.2    Sandbank, T.3    Carter, B.J.4
  • 5
    • 2642512201 scopus 로고    scopus 로고
    • Clades of adeno-associated viruses are widely disseminated in human tissues
    • Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X et al (2004) Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 78: 6381–6388
    • (2004) J Virol , vol.78 , pp. 6381-6388
    • Gao, G.1    Vandenberghe, L.H.2    Alvira, M.R.3    Lu, Y.4    Calcedo, R.5    Zhou, X.6
  • 7
    • 33748501920 scopus 로고    scopus 로고
    • Alpha2,3 and alpha2,6 N-linked sialic acids facilitate effi cient binding and transduction by adeno-associated virus types 1 and 6
    • Wu Z, Miller E, Agbandje-McKenna M, Samulski RJ (2006) Alpha2,3 and alpha2,6 N-linked sialic acids facilitate effi cient binding and transduction by adeno-associated virus types 1 and 6. J Virol 80:9093–9103
    • (2006) J Virol , vol.80 , pp. 9093-9103
    • Wu, Z.1    Miller, E.2    Agbandje-McKenna, M.3    Samulski, R.J.4
  • 8
    • 0034957168 scopus 로고    scopus 로고
    • Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and effi cient transduction but differ in sialic acid linkage specifi city
    • Kaludov N, Brown KE, Walters RW, Zabner J, Chiorini JA (2001) Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and effi cient transduction but differ in sialic acid linkage specifi city. J Virol 75:6884–6893
    • (2001) J Virol , vol.75 , pp. 6884-6893
    • Kaludov, N.1    Brown, K.E.2    Walters, R.W.3    Zabner, J.4    Chiorini, J.A.5
  • 9
    • 0031906147 scopus 로고    scopus 로고
    • Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
    • Summerford C, Samulski RJ (1998) Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72:1438–1445
    • (1998) J Virol , vol.72 , pp. 1438-1445
    • Summerford, C.1    Samulski, R.J.2
  • 10
    • 33748945422 scopus 로고    scopus 로고
    • The 37/67-kilodalton laminin receptor is a receptor for adenoassociated virus serotypes 8, 2, 3, and 9
    • Akache B, Grimm D, Pandey K, Yant SR, Xu H, Kay MA (2006) The 37/67-kilodalton laminin receptor is a receptor for adenoassociated virus serotypes 8, 2, 3, and 9. J Virol 80:9831–9836
    • (2006) J Virol , vol.80 , pp. 9831-9836
    • Akache, B.1    Grimm, D.2    Pandey, K.3    Yant, S.R.4    Xu, H.5    Kay, M.A.6
  • 12
    • 79953857433 scopus 로고    scopus 로고
    • Terminal N-linked galactose is the primary receptor for adeno-associated virus 9
    • Shen S, Bryant KD, Brown SM, Randell SH, Asokan A (2011) Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. J Biol Chem 286:13532–13540
    • (2011) J Biol Chem , vol.286 , pp. 13532-13540
    • Shen, S.1    Bryant, K.D.2    Brown, S.M.3    Randell, S.H.4    Asokan, A.5
  • 13
    • 0033010884 scopus 로고    scopus 로고
    • Human fi broblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
    • Qing K, Mah C, Hansen J, Zhou S, Dwarki V, Srivastava A (1999) Human fi broblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nat Med 5:71–77
    • (1999) Nat Med , vol.5 , pp. 71-77
    • Qing, K.1    Mah, C.2    Hansen, J.3    Zhou, S.4    Dwarki, V.5    Srivastava, A.6
  • 14
    • 0032589751 scopus 로고    scopus 로고
    • AlphaVbeta5 integrin: A co-receptor for adeno-associated virus type 2 infection
    • Summerford C, Bartlett JS, Samulski RJ (1999) AlphaVbeta5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nat Med 5:78–82
    • (1999) Nat Med , vol.5 , pp. 78-82
    • Summerford, C.1    Bartlett, J.S.2    Samulski, R.J.3
  • 15
    • 84860548973 scopus 로고    scopus 로고
    • Mutation in the platelet-derived growth factor receptor alpha inhibits adeno-associated virus type 5 transduction
    • Pilz IH, Di Pasquale G, Rzadzinska A, Leppla SH, Chiorini JA (2012) Mutation in the platelet-derived growth factor receptor alpha inhibits adeno-associated virus type 5 transduction. Virology 428:58–63
    • (2012) Virology , vol.428 , pp. 58-63
    • Pilz, I.H.1    Di Pasquale, G.2    Rzadzinska, A.3    Leppla, S.H.4    Chiorini, J.A.5
  • 16
    • 38949149695 scopus 로고    scopus 로고
    • Tropism and toxicity of adenoassociated viral vector serotypes 1, 2, 5, 6, 7, 8, and 9 in rat neurons and glia in vitro
    • Howard DB, Powers K, Wang Y, Harvey BK (2008) Tropism and toxicity of adenoassociated viral vector serotypes 1, 2, 5, 6, 7, 8, and 9 in rat neurons and glia in vitro. Virology 372:24–34
    • (2008) Virology , vol.372 , pp. 24-34
    • Howard, D.B.1    Powers, K.2    Wang, Y.3    Harvey, B.K.4
  • 17
    • 37549011375 scopus 로고    scopus 로고
    • Specifi c AAV serotypes stably transduce primary hippocampal and cortical cultures with high effi ciency and low toxicity
    • Royo NC, Vandenberghe LH, Ma JY, Hauspurg A, Yu L, Maronski M et al (2008) Specifi c AAV serotypes stably transduce primary hippocampal and cortical cultures with high effi ciency and low toxicity. Brain Res 1190:15–22
    • (2008) Brain Res , vol.1190 , pp. 15-22
    • Royo, N.C.1    Vandenberghe, L.H.2    Ma, J.Y.3    Hauspurg, A.4    Yu, L.5    Maronski, M.6
  • 18
    • 0032550793 scopus 로고    scopus 로고
    • Selective and rapid uptake of adeno-associated virus type 2 in brain
    • Bartlett JS, Samulski RJ, McCown TJ (1998) Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther 9:1181–1186
    • (1998) Hum Gene Ther , vol.9 , pp. 1181-1186
    • Bartlett, J.S.1    Samulski, R.J.2    McCown, T.J.3
  • 19
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential effi ciency and cell tropism after delivery to different regions of the central nervous system
    • Burger C, Gorbatyuk OS, Velardo MJ, Peden CS, Williams P, Zolotukhin S et al (2004) Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential effi ciency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 10:302–317
    • (2004) Mol Ther , vol.10 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3    Peden, C.S.4    Williams, P.5    Zolotukhin, S.6
  • 20
    • 0038618702 scopus 로고    scopus 로고
    • Intraventricular brain injection of adenoassociated 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-defi cient mice
    • Passini MA, Watson DJ, Vite CH, Landsburg DJ, Feigenbaum AL, Wolfe JH (2003) Intraventricular brain injection of adenoassociated 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-defi cient mice. J Virol 77:7034–7040
    • (2003) J Virol , vol.77 , pp. 7034-7040
    • Passini, M.A.1    Watson, D.J.2    Vite, C.H.3    Landsburg, D.J.4    Feigenbaum, A.L.5    Wolfe, J.H.6
  • 21
    • 32944459983 scopus 로고    scopus 로고
    • Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
    • Cearley CN, Wolfe JH (2006) Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther 13:528–537
    • (2006) Mol Ther , vol.13 , pp. 528-537
    • Cearley, C.N.1    Wolfe, J.H.2
  • 22
    • 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
    • Davidson BL, Stein CS, Heth JA, Martins I, Kotin RM, Derksen TA et al (2000) 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 U S A 97:3428–3432
    • (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 3428-3432
    • Davidson, B.L.1    Stein, C.S.2    Heth, J.A.3    Martins, I.4    Kotin, R.M.5    Derksen, T.A.6
  • 23
    • 37549061385 scopus 로고    scopus 로고
    • AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: Effects of serotype, promoter and purifi cation method
    • Klein RL, Dayton RD, Tatom JB, Henderson KM, Henning PP (2008) AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purifi cation method. Mol Ther 16:89–96
    • (2008) Mol Ther , vol.16 , pp. 89-96
    • Klein, R.L.1    Dayton, R.D.2    Tatom, J.B.3    Henderson, K.M.4    Henning, P.P.5
  • 24
    • 84855453499 scopus 로고    scopus 로고
    • Corticospinal tract transduction: A comparison of seven adeno- associated viral vector serotypes and a non-integrating lentiviral vector
    • Hutson TH, Verhaagen J, Yanez-Munoz RJ, Moon LD (2012) Corticospinal tract transduction: a comparison of seven adeno- associated viral vector serotypes and a non-integrating lentiviral vector. Gene Ther 19:49–60
    • (2012) Gene Ther , vol.19 , pp. 49-60
    • Hutson, T.H.1    Verhaagen, J.2    Yanez-Munoz, R.J.3    Moon, L.D.4
  • 25
    • 26844444953 scopus 로고    scopus 로고
    • Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS
    • Liu G, Martins IH, Chiorini JA, Davidson BL (2005) Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS. Gene Ther 12:1503–1508
    • (2005) Gene Ther , vol.12 , pp. 1503-1508
    • Liu, G.1    Martins, I.H.2    Chiorini, J.A.3    Davidson, B.L.4
  • 26
    • 70349850388 scopus 로고    scopus 로고
    • Effi cient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
    • Lawlor PA, Bland RJ, Mouravlev A, Young D, During MJ (2009) Effi cient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol Ther 17:1692–1702
    • (2009) Mol Ther , vol.17 , pp. 1692-1702
    • Lawlor, P.A.1    Bland, R.J.2    Mouravlev, A.3    Young, D.4    During, M.J.5
  • 27
    • 32944471565 scopus 로고    scopus 로고
    • Effi cient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fl uorescent proteins
    • Klein RL, Dayton RD, Leidenheimer NJ, Jansen K, Golde TE, Zweig RM (2006) Effi cient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fl uorescent proteins. Mol Ther 13:517–527
    • (2006) Mol Ther , vol.13 , pp. 517-527
    • Klein, R.L.1    Dayton, R.D.2    Leidenheimer, N.J.3    Jansen, K.4    Golde, T.E.5    Zweig, R.M.6
  • 28
    • 34547118339 scopus 로고    scopus 로고
    • Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat
    • Reimsnider S, Manfredsson FP, Muzyczka N, Mandel RJ (2007) Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat. Mol Ther 15:1504–1511
    • (2007) Mol Ther , vol.15 , pp. 1504-1511
    • Reimsnider, S.1    Manfredsson, F.P.2    Muzyczka, N.3    Mandel, R.J.4
  • 30
    • 33847211798 scopus 로고    scopus 로고
    • Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 Rhesus macaque-derived adeno-associated virus vector
    • Sondhi D, Hackett NR, Peterson DA, Stratton J, Baad M, Travis KM et al (2007) Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 Rhesus macaque-derived adeno-associated virus vector. Mol Ther 15:481–491
    • (2007) Mol Ther , vol.15 , pp. 481-491
    • Sondhi, D.1    Hackett, N.R.2    Peterson, D.A.3    Stratton, J.4    Baad, M.5    Travis, K.M.6
  • 31
    • 0141996932 scopus 로고    scopus 로고
    • Adeno-associated virus vectormediated transduction in the cat brain
    • Vite CH, Passini MA, Haskins ME, Wolfe JH (2003) Adeno-associated virus vectormediated transduction in the cat brain. Gene Ther 10:1874–1881
    • (2003) Gene Ther , vol.10 , pp. 1874-1881
    • Vite, C.H.1    Passini, M.A.2    Haskins, M.E.3    Wolfe, J.H.4
  • 32
    • 1542529998 scopus 로고    scopus 로고
    • Gene delivery to the mouse brain with adeno- associated virus
    • Passini MA, Watson DJ, Wolfe JH (2004) Gene delivery to the mouse brain with adeno- associated virus. Methods Mol Biol 246:225–236
    • (2004) Methods Mol Biol , vol.246 , pp. 225-236
    • Passini, M.A.1    Watson, D.J.2    Wolfe, J.H.3
  • 33
    • 80051548392 scopus 로고    scopus 로고
    • Comparison of AAV2 and AAV5 in gene transfer in the injured spinal cord of mice
    • Peng SP, Kugler S, Ma ZK, Shen YQ, Schachner M (2011) Comparison of AAV2 and AAV5 in gene transfer in the injured spinal cord of mice. Neuroreport 22:565–569
    • (2011) Neuroreport , vol.22 , pp. 565-569
    • Peng, S.P.1    Kugler, S.2    Ma, Z.K.3    Shen, Y.Q.4    Schachner, M.5
  • 34
    • 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
    • Cearley CN, Wolfe JH (2007) 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 27:9928–9940
    • (2007) J Neurosci , vol.27 , pp. 9928-9940
    • Cearley, C.N.1    Wolfe, J.H.2
  • 35
    • 33750687840 scopus 로고    scopus 로고
    • Intra-ventricular infusion of rAAV1-EGFP resulted in transduction in multiple regions of adult rat brain: A comparative study with rAAV2 and rAAV5 vectors
    • Li SF, Wang RZ, Meng QH, Li GL, Hu GJ, Dou WC et al (2006) Intra-ventricular infusion of rAAV1-EGFP resulted in transduction in multiple regions of adult rat brain: a comparative study with rAAV2 and rAAV5 vectors. Brain Res 1122:1–9
    • (2006) Brain Res , vol.1122 , pp. 1-9
    • Li, S.F.1    Wang, R.Z.2    Meng, Q.H.3    Li, G.L.4    Hu, G.J.5    Dou, W.C.6
  • 36
    • 54749126318 scopus 로고    scopus 로고
    • Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
    • Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH (2008) Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther 16:1710–1718
    • (2008) Mol Ther , vol.16 , pp. 1710-1718
    • Cearley, C.N.1    Vandenberghe, L.H.2    Parente, M.K.3    Carnish, E.R.4    Wilson, J.M.5    Wolfe, J.H.6
  • 37
    • 84875925195 scopus 로고    scopus 로고
    • Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in nonhuman primates
    • Gray SJ, Nagabhushan KS, McCown TJ, Jude SR (2013) Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in nonhuman primates. Gene Ther 20:450–459
    • (2013) Gene Ther , vol.20 , pp. 450-459
    • Gray, S.J.1    Nagabhushan, K.S.2    McCown, T.J.3    Jude, S.R.4
  • 38
    • 84877847142 scopus 로고    scopus 로고
    • Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fl uid of nonhuman primates
    • Samaranch L, Salegio EA, San SW, Kells AP, Bringas JR, Forsayeth J et al (2013) Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fl uid of nonhuman primates. Hum Gene Ther 24:526–532
    • (2013) Hum Gene Ther , vol.24 , pp. 526-532
    • Samaranch, L.1    Salegio, E.A.2    San, S.W.3    Kells, A.P.4    Bringas, J.R.5    Forsayeth, J.6
  • 39
    • 84859094442 scopus 로고    scopus 로고
    • AAV8(Gfp) preferentially targets large diameter dorsal root ganglion neurones after both intra-dorsal root ganglion and intrathecal injection
    • Jacques SJ, Ahmed Z, Forbes A, Douglas MR, Vigenswara V, Berry M et al (2012) AAV8(gfp) preferentially targets large diameter dorsal root ganglion neurones after both intra-dorsal root ganglion and intrathecal injection. Mol Cell Neurosci 49:464–474
    • (2012) Mol Cell Neurosci , vol.49 , pp. 464-474
    • Jacques, S.J.1    Ahmed, Z.2    Forbes, A.3    Douglas, M.R.4    Vigenswara, V.5    Berry, M.6
  • 40
    • 80053009250 scopus 로고    scopus 로고
    • Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery
    • Snyder BR, Gray SJ, Quach ET, Huang JW, Leung CH, Samulski RJ et al (2011) Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum Gene Ther 22:1129–1135
    • (2011) Hum Gene Ther , vol.22 , pp. 1129-1135
    • Snyder, B.R.1    Gray, S.J.2    Quach, E.T.3    Huang, J.W.4    Leung, C.H.5    Samulski, R.J.6
  • 42
    • 79957888016 scopus 로고    scopus 로고
    • Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates
    • Gray SJ, Matagne V, Bachaboina L, Yadav S, Ojeda SR, Samulski RJ (2011) Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther 19:1058–1069
    • (2011) Mol Ther , vol.19 , pp. 1058-1069
    • Gray, S.J.1    Matagne, V.2    Bachaboina, L.3    Yadav, S.4    Ojeda, S.R.5    Samulski, R.J.6
  • 43
    • 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
    • Yang B, Li S, Wang H, Guo Y, Gessler DJ, Cao C et al (2014) Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther 22:1299–1309
    • (2014) Mol Ther , vol.22 , pp. 1299-1309
    • Yang, B.1    Li, S.2    Wang, H.3    Guo, Y.4    Gessler, D.J.5    Cao, C.6
  • 44
    • 79961032418 scopus 로고    scopus 로고
    • Several rAAV vectors effi - ciently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    • Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R et al (2011) Several rAAV vectors effi - ciently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 19:1440–1448
    • (2011) Mol Ther , vol.19 , pp. 1440-1448
    • Zhang, H.1    Yang, B.2    Mu, X.3    Ahmed, S.S.4    Su, Q.5    He, R.6
  • 45
    • 44349169012 scopus 로고    scopus 로고
    • Systemic AAV6 delivery mediating RNA interference against SOD1: Neuromuscular transduction does not alter disease progression in fALS mice
    • Towne C, Raoul C, Schneider BL, Aebischer P (2008) Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol Ther 16:1018–1025
    • (2008) Mol Ther , vol.16 , pp. 1018-1025
    • Towne, C.1    Raoul, C.2    Schneider, B.L.3    Aebischer, P.4
  • 46
    • 0347286956 scopus 로고    scopus 로고
    • Selfcomplementary adeno-associated virus serotype 2 vector: Global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain
    • Fu H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X et al (2003) Selfcomplementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 8:911–917
    • (2003) Mol Ther , vol.8 , pp. 911-917
    • Fu, H.1    Muenzer, J.2    Samulski, R.J.3    Breese, G.4    Sifford, J.5    Zeng, X.6
  • 47
    • 84901447384 scopus 로고    scopus 로고
    • Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia
    • Chen SJ, Sanmiguel J, Lock M, McMenamin D, Draper C, Limberis MP et al (2013) Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia. Hum Gene Ther Clin Dev 24:154–160
    • (2013) Hum Gene Ther Clin Dev , vol.24 , pp. 154-160
    • Chen, S.J.1    Sanmiguel, J.2    Lock, M.3    McMenamin, D.4    Draper, C.5    Limberis, M.P.6
  • 48
    • 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
    • Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J et al (2005) 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 12:1072–1082
    • (2005) Mol Ther , vol.12 , pp. 1072-1082
    • Acland, G.M.1    Aguirre, G.D.2    Bennett, J.3    Aleman, T.S.4    Cideciyan, A.V.5    Bennicelli, J.6
  • 49
    • 33846933945 scopus 로고    scopus 로고
    • Restoration of vision in RPE65-defi cient Briard dogs using an AAV serotype 4 vector that specifi cally targets the retinal pigmented epithelium
    • Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D et al (2007) Restoration of vision in RPE65-defi cient Briard dogs using an AAV serotype 4 vector that specifi cally targets the retinal pigmented epithelium. Gene Ther 14:292–303
    • (2007) Gene Ther , vol.14 , pp. 292-303
    • Le Meur, G.1    Stieger, K.2    Smith, A.J.3    Weber, M.4    Deschamps, J.Y.5    Nivard, D.6
  • 50
    • 42549166922 scopus 로고    scopus 로고
    • Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain
    • Stieger K, Colle MA, Dubreil L, Mendes- Madeira A, Weber M, Le Meur G et al (2008) Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol Ther 16:916–923
    • (2008) Mol Ther , vol.16 , pp. 916-923
    • Stieger, K.1    Colle, M.A.2    Dubreil, L.3    Mendes- Madeira, A.4    Weber, M.5    Le Meur, G.6
  • 52
    • 67651097445 scopus 로고    scopus 로고
    • Adeno-associated virus serotype-9 effi ciently transduces the retinal outer plexiform layer
    • Lei B, Zhang K, Yue Y, Ghosh A, Duan D (2009) Adeno-associated virus serotype-9 effi ciently transduces the retinal outer plexiform layer. Mol Vis 15:1374–1382
    • (2009) Mol Vis , vol.15 , pp. 1374-1382
    • Lei, B.1    Zhang, K.2    Yue, Y.3    Ghosh, A.4    Duan, D.5
  • 55
    • 84873829632 scopus 로고    scopus 로고
    • The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specifi c expression in the primate retina
    • Boye SE, Alexander JJ, Boye SL, Witherspoon CD, Sandefer KJ, Conlon TJ et al (2012) The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specifi c expression in the primate retina. Hum Gene Ther 23:1101–1115
    • (2012) Hum Gene Ther , vol.23 , pp. 1101-1115
    • Boye, S.E.1    Alexander, J.J.2    Boye, S.L.3    Witherspoon, C.D.4    Sandefer, K.J.5    Conlon, T.J.6
  • 56
    • 38649139370 scopus 로고    scopus 로고
    • Effi cient retrograde neuronal transduction utilizing selfcomplementary AAV1
    • Hollis ER 2nd, Kadoya K, Hirsch M, Samulski RJ, Tuszynski MH (2008) Effi cient retrograde neuronal transduction utilizing selfcomplementary AAV1. Mol Ther 16:296–301
    • (2008) Mol Ther , vol.16 , pp. 296-301
    • Hollis, E.R.1    Kadoya, K.2    Hirsch, M.3    Samulski, R.J.4    Tuszynski, M.H.5
  • 58
    • 84895461561 scopus 로고    scopus 로고
    • Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is traffi cked in a highly motile Rab7-positive compartment
    • Castle MJ, Perlson E, Holzbaur EL, Wolfe JH (2014) Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is traffi cked in a highly motile Rab7-positive compartment. Mol Ther 22:554–566
    • (2014) Mol Ther , vol.22 , pp. 554-566
    • Castle, M.J.1    Perlson, E.2    Holzbaur, E.L.3    Wolfe, J.H.4
  • 59
    • 84880338159 scopus 로고    scopus 로고
    • Intraspinal AAV injections immediately rostral to a thoracic spinal cord injury site effi ciently transduces neurons in spinal cord and brain
    • Klaw MC, Xu C, Tom VJ (2013) Intraspinal AAV injections immediately rostral to a thoracic spinal cord injury site effi ciently transduces neurons in spinal cord and brain. Mol Ther Nucleic Acids 2, e108
    • (2013) Mol Ther Nucleic Acids , vol.2
    • Klaw, M.C.1    Xu, C.2    Tom, V.J.3
  • 60
    • 79957686690 scopus 로고    scopus 로고
    • Effi cient gene transfer into neurons in monkey brain by adeno-associated virus 8
    • Masamizu Y, Okada T, Ishibashi H, Takeda S, Yuasa S, Nakahara K (2010) Effi cient gene transfer into neurons in monkey brain by adeno-associated virus 8. Neuroreport 21:447–451
    • (2010) Neuroreport , vol.21 , pp. 447-451
    • Masamizu, Y.1    Okada, T.2    Ishibashi, H.3    Takeda, S.4    Yuasa, S.5    Nakahara, K.6
  • 61
    • 50249125921 scopus 로고    scopus 로고
    • Molecular engineering of viral gene delivery vehicles
    • Schaffer DV, Koerber JT, Lim KI (2008) Molecular engineering of viral gene delivery vehicles. Annu Rev Biomed Eng 10:169–194
    • (2008) Annu Rev Biomed Eng , vol.10 , pp. 169-194
    • Schaffer, D.V.1    Koerber, J.T.2    Lim, K.I.3
  • 62
    • 79960466427 scopus 로고    scopus 로고
    • Receptors and tropisms of envelope viruses
    • Morizono K, Chen IS (2011) Receptors and tropisms of envelope viruses. Curr Opin Virol 1:13–18
    • (2011) Curr Opin Virol , vol.1 , pp. 13-18
    • Morizono, K.1    Chen, I.S.2
  • 63
    • 0033007934 scopus 로고    scopus 로고
    • Targeted adenoassocia ted virus vector transduction of nonpermissive cells mediated by a bispecifi c F(Ab’gamma)2 antibody
    • Bartlett JS, Kleinschmidt J, Boucher RC, Samulski RJ (1999) Targeted adenoassocia ted virus vector transduction of nonpermissive cells mediated by a bispecifi c F(ab’gamma)2 antibody. Nat Biotechnol 17:181–186
    • (1999) Nat Biotechnol , vol.17 , pp. 181-186
    • Bartlett, J.S.1    Kleinschmidt, J.2    Boucher, R.C.3    Samulski, R.J.4
  • 64
    • 0036893196 scopus 로고    scopus 로고
    • Conjugate-based targeting of recombinant adeno-associated virus type 2 vectors by using avidin-linked ligands
    • Ponnazhagan S, Mahendra G, Kumar S, Thompson JA, Castillas M Jr (2002) Conjugate-based targeting of recombinant adeno-associated virus type 2 vectors by using avidin-linked ligands. J Virol 76: 12900–12907
    • (2002) J Virol , vol.76 , pp. 12900-12907
    • Ponnazhagan, S.1    Mahendra, G.2    Kumar, S.3    Thompson, J.A.4    Castillas, M.5
  • 65
    • 26944480735 scopus 로고    scopus 로고
    • Utility of PEGylated recombinant adeno-associated viruses for gene transfer
    • Le HT, Yu QC, Wilson JM, Croyle MA (2005) Utility of PEGylated recombinant adeno-associated viruses for gene transfer. J Control Release 108:161–177
    • (2005) J Control Release , vol.108 , pp. 161-177
    • Le, H.T.1    Yu, Q.C.2    Wilson, J.M.3    Croyle, M.A.4
  • 66
    • 42049117918 scopus 로고    scopus 로고
    • Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera
    • Carlisle RC, Benjamin R, Briggs SS, Sumner- Jones S, McIntosh J, Gill D et al (2008) Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera. J Gene Med 10:400–411
    • (2008) J Gene Med , vol.10 , pp. 400-411
    • Carlisle, R.C.1    Benjamin, R.2    Briggs, S.S.3    Sumner- Jones, S.4    McIntosh, J.5    Gill, D.6
  • 67
    • 79955034766 scopus 로고    scopus 로고
    • Glycated AAV vectors: Chemical redirection of viral tissue tropism
    • Horowitz ED, Weinberg MS, Asokan A (2011) Glycated AAV vectors: chemical redirection of viral tissue tropism. Bioconjug Chem 22:529–532
    • (2011) Bioconjug Chem , vol.22 , pp. 529-532
    • Horowitz, E.D.1    Weinberg, M.S.2    Asokan, A.3
  • 68
    • 0034531297 scopus 로고    scopus 로고
    • Building a better vector: The manipulation of AAV virions
    • Rabinowitz JE, Samulski RJ (2000) Building a better vector: the manipulation of AAV virions. Virology 278:301–308
    • (2000) Virology , vol.278 , pp. 301-308
    • Rabinowitz, J.E.1    Samulski, R.J.2
  • 69
    • 0037368410 scopus 로고    scopus 로고
    • Generation and characterization of chimeric recombinant AAV vectors
    • Hauck B, Chen L, Xiao W (2003) Generation and characterization of chimeric recombinant AAV vectors. Mol Ther 7:419–425
    • (2003) Mol Ther , vol.7 , pp. 419-425
    • Hauck, B.1    Chen, L.2    Xiao, W.3
  • 70
    • 77958605778 scopus 로고    scopus 로고
    • Expression of human A53T alpha-synuclein in the rat substantia nigra using a novel AAV1/2 vector produces a rapidly evolving pathology with protein aggregation, dystrophic neurite architecture and nigrostriatal degeneration with potential to model the pathology of Parkinson’s disease
    • Koprich JB, Johnston TH, Reyes MG, Sun X, Brotchie JM (2010) Expression of human A53T alpha-synuclein in the rat substantia nigra using a novel AAV1/2 vector produces a rapidly evolving pathology with protein aggregation, dystrophic neurite architecture and nigrostriatal degeneration with potential to model the pathology of Parkinson’s disease. Mol Neurodegener 5:43
    • (2010) Mol Neurodegener , vol.5 , pp. 43
    • Koprich, J.B.1    Johnston, T.H.2    Reyes, M.G.3    Sun, X.4    Brotchie, J.M.5
  • 71
    • 4444352441 scopus 로고    scopus 로고
    • Cross-dressing the virion: The transcapsidation of adeno-associated virus serotypes functionally defi nes subgroups
    • Rabinowitz JE, Bowles DE, Faust SM, Ledford JG, Cunningham SE, Samulski RJ (2004) Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defi nes subgroups. J Virol 78:4421–4432
    • (2004) J Virol , vol.78 , pp. 4421-4432
    • Rabinowitz, J.E.1    Bowles, D.E.2    Faust, S.M.3    Ledford, J.G.4    Cunningham, S.E.5    Samulski, R.J.6
  • 72
    • 0032170198 scopus 로고    scopus 로고
    • Development of novel cell surface CD34-targeted recombinant adenoassociated virus vectors for gene therapy
    • Yang Q, Mamounas M, Yu G, Kennedy S, Leaker B, Merson J et al (1998) Development of novel cell surface CD34-targeted recombinant adenoassociated virus vectors for gene therapy. Hum Gene Ther 9:1929–1937
    • (1998) Hum Gene Ther , vol.9 , pp. 1929-1937
    • Yang, Q.1    Mamounas, M.2    Yu, G.3    Kennedy, S.4    Leaker, B.5    Merson, J.6
  • 73
    • 0038618725 scopus 로고    scopus 로고
    • Identifi cation of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding
    • Opie SR, Warrington KH, Agbandje- McKenna M, Zolotukhin S, Muzyczka N (2003) Identifi cation of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding. J Virol 77:6995–7006
    • (2003) J Virol , vol.77 , pp. 6995-7006
    • Opie, S.R.1    Warrington, K.H.2    Agbandje- McKenna, M.3    Zolotukhin, S.4    Muzyczka, N.5
  • 74
    • 0032870158 scopus 로고    scopus 로고
    • Genetic capsid modifi cations allow effi cient re-targeting of adeno-associated virus type 2
    • Girod A, Ried M, Wobus C, Lahm H, Leike K, Kleinschmidt J et al (1999) Genetic capsid modifi cations allow effi cient re-targeting of adeno-associated virus type 2. Nat Med 5:1438
    • (1999) Nat Med , vol.5 , pp. 1438
    • Girod, A.1    Ried, M.2    Wobus, C.3    Lahm, H.4    Leike, K.5    Kleinschmidt, J.6
  • 75
    • 0034814413 scopus 로고    scopus 로고
    • Insertional mutagenesis of the adenoassociated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors
    • Shi W, Arnold GS, Bartlett JS (2001) Insertional mutagenesis of the adenoassociated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors targeted to alternative cell-surface receptors. Hum Gene Ther 12:1697–1711
    • (2001) Hum Gene Ther , vol.12 , pp. 1697-1711
    • Shi, W.1    Arnold, G.S.2    Bartlett, J.S.3
  • 76
    • 0033851104 scopus 로고    scopus 로고
    • Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism
    • Wu P, Xiao W, Conlon T, Hughes J, Agbandje- McKenna M, Ferkol T et al (2000) Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol 74:8635–8647
    • (2000) J Virol , vol.74 , pp. 8635-8647
    • Wu, P.1    Xiao, W.2    Conlon, T.3    Hughes, J.4    Agbandje- McKenna, M.5    Ferkol, T.6
  • 77
    • 0034963928 scopus 로고    scopus 로고
    • Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids
    • Grifman M, Trepel M, Speece P, Gilbert LB, Arap W, Pasqualini R et al (2001) Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids. Mol Ther 3:964–975
    • (2001) Mol Ther , vol.3 , pp. 964-975
    • Grifman, M.1    Trepel, M.2    Speece, P.3    Gilbert, L.B.4    Arap, W.5    Pasqualini, R.6
  • 78
  • 80
    • 84869390793 scopus 로고    scopus 로고
    • Random insertion of mCherry Into VP3 domain of adeno-associated virus yields fl uorescent capsids with no loss of infectivity
    • Judd J, Wei F, Nguyen PQ, Tartaglia LJ, Agbandje-McKenna M, Silberg JJ et al (2012) Random insertion of mCherry Into VP3 domain of adeno-associated virus yields fl uorescent capsids with no loss of infectivity. Mol Ther Nucleic Acids 1:e54
    • (2012) Mol Ther Nucleic Acids , vol.1
    • Judd, J.1    Wei, F.2    Nguyen, P.Q.3    Tartaglia, L.J.4    Agbandje-McKenna, M.5    Silberg, J.J.6
  • 81
    • 69249213448 scopus 로고    scopus 로고
    • A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery
    • Yu CY, Yuan Z, Cao Z, Wang B, Qiao C, Li J et al (2009) A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery. Gene Ther 16:953–962
    • (2009) Gene Ther , vol.16 , pp. 953-962
    • Yu, C.Y.1    Yuan, Z.2    Cao, Z.3    Wang, B.4    Qiao, C.5    Li, J.6
  • 83
    • 77955654722 scopus 로고    scopus 로고
    • Hearttargeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
    • Ying Y, Muller OJ, Goehringer C, Leuchs B, Trepel M, Katus HA et al (2010) Hearttargeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library. Gene Ther 17:980–990
    • (2010) Gene Ther , vol.17 , pp. 980-990
    • Ying, Y.1    Muller, O.J.2    Goehringer, C.3    Leuchs, B.4    Trepel, M.5    Katus, H.A.6
  • 84
    • 0842264162 scopus 로고    scopus 로고
    • Targeted gene delivery to vascular tissue in vivo by tropism-modifi ed adeno-associated virus vectors
    • White SJ, Nicklin SA, Buning H, Brosnan MJ, Leike K, Papadakis ED et al (2004) Targeted gene delivery to vascular tissue in vivo by tropism-modifi ed adeno-associated virus vectors. Circulation 109:513–519
    • (2004) Circulation , vol.109 , pp. 513-519
    • White, S.J.1    Nicklin, S.A.2    Buning, H.3    Brosnan, M.J.4    Leike, K.5    Papadakis, E.D.6
  • 85
    • 33645502138 scopus 로고    scopus 로고
    • Vascular bed-targeted in vivo gene delivery using tropism- modifi ed adeno-associated viruses
    • Work LM, Buning H, Hunt E, Nicklin SA, Denby L, Britton N et al (2006) Vascular bed-targeted in vivo gene delivery using tropism- modifi ed adeno-associated viruses. Mol Ther 13:683–693
    • (2006) Mol Ther , vol.13 , pp. 683-693
    • Work, L.M.1    Buning, H.2    Hunt, E.3    Nicklin, S.A.4    Denby, L.5    Britton, N.6
  • 86
    • 40249103201 scopus 로고    scopus 로고
    • Engineering adeno-associated virus 2 vectors for targeted gene delivery to atherosclerotic lesions
    • White K, Buning H, Kritz A, Janicki H, McVey J, Perabo L et al (2008) Engineering adeno-associated virus 2 vectors for targeted gene delivery to atherosclerotic lesions. Gene Ther 15:443–451
    • (2008) Gene Ther , vol.15 , pp. 443-451
    • White, K.1    Buning, H.2    Kritz, A.3    Janicki, H.4    McVey, J.5    Perabo, L.6
  • 87
    • 65249116937 scopus 로고    scopus 로고
    • Successful expansion but not complete restriction of tropism of adenoassociated virus by in vivo biopanning of random virus display peptide libraries
    • Michelfelder S, Kohlschutter J, Skorupa A, Pfennings S, Muller O, Kleinschmidt JA et al (2009) Successful expansion but not complete restriction of tropism of adenoassociated virus by in vivo biopanning of random virus display peptide libraries. PLoS One 4:e5122
    • (2009) Plos One , vol.4
    • Michelfelder, S.1    Kohlschutter, J.2    Skorupa, A.3    Pfennings, S.4    Muller, O.5    Kleinschmidt, J.A.6
  • 88
    • 79961162172 scopus 로고    scopus 로고
    • Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo
    • Michelfelder S, Varadi K, Raupp C, Hunger A, Korbelin J, Pahrmann C et al (2011) Peptide ligands incorporated into the threefold spike capsid domain to re-direct gene transduction of AAV8 and AAV9 in vivo. PLoS One 6:e23101
    • (2011) Plos One , vol.6
    • Michelfelder, S.1    Varadi, K.2    Raupp, C.3    Hunger, A.4    Korbelin, J.5    Pahrmann, C.6
  • 89
    • 70350502352 scopus 로고    scopus 로고
    • Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
    • Chen YH, Chang M, Davidson BL (2009) Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy. Nat Med 15:1215–1218
    • (2009) Nat Med , vol.15 , pp. 1215-1218
    • Chen, Y.H.1    Chang, M.2    Davidson, B.L.3
  • 90
    • 84880540708 scopus 로고    scopus 로고
    • Vivodirected evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous
    • Dalkara D, Byrne LC, Klimczak RR, Visel M, Yin L, Merigan WH et al (2013) In vivodirected evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med 5:189ra176
    • (2013) Sci Transl Med , vol.5
    • Dalkara, D.1    Byrne, L.C.2    Klimczak, R.R.3    Visel, M.4    Yin, L.5    Merigan, W.H.6
  • 91
    • 0038027345 scopus 로고    scopus 로고
    • RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfateindependent cell entry mechanism
    • Shi W, Bartlett JS (2003) RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfateindependent cell entry mechanism. Mol Ther 7:515–525
    • (2003) Mol Ther , vol.7 , pp. 515-525
    • Shi, W.1    Bartlett, J.S.2
  • 92
    • 0141454787 scopus 로고    scopus 로고
    • Identifi cation of a heparin-binding motif on adeno-associated virus type 2 capsids
    • Kern A, Schmidt K, Leder C, Muller OJ, Wobus CE, Bettinger K et al (2003) Identifi cation of a heparin-binding motif on adeno-associated virus type 2 capsids. J Virol 77:11072–11081
    • (2003) J Virol , vol.77 , pp. 11072-11081
    • Kern, A.1    Schmidt, K.2    Leder, C.3    Muller, O.J.4    Wobus, C.E.5    Bettinger, K.6
  • 93
    • 45749099260 scopus 로고    scopus 로고
    • Engineering and selection of shuffl ed AAV genomes: A new strategy for producing targeted biological nanoparticles
    • Li W, Asokan A, Wu Z, Van Dyke T, DiPrimio N, Johnson JS et al (2008) Engineering and selection of shuffl ed AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther 16:1252–1260
    • (2008) Mol Ther , vol.16 , pp. 1252-1260
    • Li, W.1    Asokan, A.2    Wu, Z.3    Van Dyke, T.4    Diprimio, N.5    Johnson, J.S.6
  • 94
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adenoassociated virus yields enhanced gene delivery vectors
    • Maheshri N, Koerber JT, Kaspar BK, Schaffer DV (2006) Directed evolution of adenoassociated virus yields enhanced gene delivery vectors. Nat Biotechnol 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
  • 95
    • 62649151797 scopus 로고    scopus 로고
    • A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffl ing and in vivo selection
    • Yang L, Jiang J, Drouin LM, Agbandje- McKenna M, Chen C, Qiao C et al (2009) A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffl ing and in vivo selection. Proc Natl Acad Sci U S A 106: 3946–3951
    • (2009) Proc Natl Acad Sci U S A , vol.106 , pp. 3946-3951
    • Yang, L.1    Jiang, J.2    Drouin, L.M.3    Agbandje- McKenna, M.4    Chen, C.5    Qiao, C.6
  • 97
    • 73849130017 scopus 로고    scopus 로고
    • Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium
    • Li W, Zhang L, Johnson JS, Zhijian W, Grieger JC, Ping-Jie X et al (2009) Generation of novel AAV variants by directed evolution for improved CFTR delivery to human ciliated airway epithelium. Mol Ther 17: 2067–2077
    • (2009) Mol Ther , vol.17 , pp. 2067-2077
    • Li, W.1    Zhang, L.2    Johnson, J.S.3    Zhijian, W.4    Grieger, J.C.5    Ping-Jie, X.6
  • 98
    • 70449338146 scopus 로고    scopus 로고
    • A novel adeno-associated viral variant for effi cient and selective intravitreal transduction of rat Muller cells
    • Klimczak RR, Koerber JT, Dalkara D, Flannery JG, Schaffer DV (2009) A novel adeno-associated viral variant for effi cient and selective intravitreal transduction of rat Muller cells. PLoS One 4:e7467
    • (2009) Plos One , vol.4
    • Klimczak, R.R.1    Koerber, J.T.2    Dalkara, D.3    Flannery, J.G.4    Schaffer, D.V.5
  • 101
    • 77649273563 scopus 로고    scopus 로고
    • Directed evolution of a novel adenoassociated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB)
    • Gray SJ, Blake BL, Criswell HE, Nicolson SC, Samulski RJ, McCown TJ et al (2010) Directed evolution of a novel adenoassociated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). Mol Ther 18:570–578
    • (2010) Mol Ther , vol.18 , pp. 570-578
    • Gray, S.J.1    Blake, B.L.2    Criswell, H.E.3    Nicolson, S.C.4    Samulski, R.J.5    McCown, T.J.6
  • 102
    • 79953325690 scopus 로고    scopus 로고
    • An evolved adenoassociated viral variant enhances gene delivery and gene targeting in neural stem cells
    • Jang JH, Koerber JT, Kim JS, Asuri P, Vazin T, Bartel M et al (2011) An evolved adenoassociated viral variant enhances gene delivery and gene targeting in neural stem cells. Mol Ther 19:667–675
    • (2011) Mol Ther , vol.19 , pp. 667-675
    • Jang, J.H.1    Koerber, J.T.2    Kim, J.S.3    Asuri, P.4    Vazin, T.5    Bartel, M.6
  • 103
    • 84856546741 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells
    • Asuri P, Bartel MA, Vazin T, Jang JH, Wong TB, Schaffer DV (2012) Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells. Mol Ther 20:329–338
    • (2012) Mol Ther , vol.20 , pp. 329-338
    • Asuri, P.1    Bartel, M.A.2    Vazin, T.3    Jang, J.H.4    Wong, T.B.5    Schaffer, D.V.6
  • 104
    • 74049120342 scopus 로고    scopus 로고
    • Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
    • Asokan A, Conway JC, Phillips JL, Li C, Hegge J, Sinnott R et al (2010) Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat Biotechnol 28:79–82
    • (2010) Nat Biotechnol , vol.28 , pp. 79-82
    • Asokan, A.1    Conway, J.C.2    Phillips, J.L.3    Li, C.4    Hegge, J.5    Sinnott, R.6
  • 105
    • 84856515432 scopus 로고    scopus 로고
    • Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector
    • Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS et al (2012) Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther 20:443–455
    • (2012) Mol Ther , vol.20 , pp. 443-455
    • Bowles, D.E.1    McPhee, S.W.2    Li, C.3    Gray, S.J.4    Samulski, J.J.5    Camp, A.S.6
  • 106
    • 33750722840 scopus 로고    scopus 로고
    • Single amino acid changes can infl uence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes
    • Wu Z, Asokan A, Grieger JC, Govindasamy L, Agbandje-McKenna M, Samulski RJ (2006) Single amino acid changes can infl uence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol 80:11393–11397
    • (2006) J Virol , vol.80 , pp. 11393-11397
    • Wu, Z.1    Asokan, A.2    Grieger, J.C.3    Govindasamy, L.4    Agbandje-McKenna, M.5    Samulski, R.J.6
  • 107
    • 73849120718 scopus 로고    scopus 로고
    • Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints
    • Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, Sandhu A et al (2009) Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther 16:1416–1428
    • (2009) Gene Ther , vol.16 , pp. 1416-1428
    • Vandenberghe, L.H.1    Breous, E.2    Nam, H.J.3    Gao, G.4    Xiao, R.5    Sandhu, A.6
  • 108
    • 62549150024 scopus 로고    scopus 로고
    • Transduction effi ciencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro
    • Limberis MP, Vandenberghe LH, Zhang L, Pickles RJ, Wilson JM (2009) Transduction effi ciencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol Ther 17:294–301
    • (2009) Mol Ther , vol.17 , pp. 294-301
    • Limberis, M.P.1    Vandenberghe, L.H.2    Zhang, L.3    Pickles, R.J.4    Wilson, J.M.5
  • 109
    • 30344450073 scopus 로고    scopus 로고
    • Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization
    • Lochrie MA, Tatsuno GP, Christie B, McDonnell JW, Zhou S, Surosky R et al (2006) Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol 80:821–834
    • (2006) J Virol , vol.80 , pp. 821-834
    • Lochrie, M.A.1    Tatsuno, G.P.2    Christie, B.3    McDonnell, J.W.4    Zhou, S.5    Surosky, R.6
  • 110
    • 84864390047 scopus 로고    scopus 로고
    • Single amino acid modifi cation of adeno-associated virus capsid changes transduction and humoral immune profi les
    • Li C, Diprimio N, Bowles DE, Hirsch ML, Monahan PE, Asokan A et al (2012) Single amino acid modifi cation of adeno-associated virus capsid changes transduction and humoral immune profi les. J Virol 86:7752–7759
    • (2012) J Virol , vol.86 , pp. 7752-7759
    • Li, C.1    Diprimio, N.2    Bowles, D.E.3    Hirsch, M.L.4    Monahan, P.E.5    Asokan, A.6
  • 111
    • 45549090635 scopus 로고    scopus 로고
    • Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-effi ciency transduction at lower doses
    • Zhong L, Li B, Mah CS, Govindasamy L, Agbandje-McKenna M, Cooper M et al (2008) Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-effi ciency transduction at lower doses. Proc Natl Acad Sci U S A 105: 7827–7832
    • (2008) Proc Natl Acad Sci U S A , vol.105 , pp. 7827-7832
    • Zhong, L.1    Li, B.2    Mah, C.S.3    Govindasamy, L.4    Agbandje-McKenna, M.5    Cooper, M.6
  • 112
    • 84876860169 scopus 로고    scopus 로고
    • Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors
    • Kay CN, Ryals RC, Aslanidi GV, Min SH, Ruan Q, Sun J et al (2013) Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One 8: e62097
    • (2013) Plos One , vol.8
    • Kay, C.N.1    Ryals, R.C.2    Aslanidi, G.V.3    Min, S.H.4    Ruan, Q.5    Sun, J.6
  • 113
    • 78649375117 scopus 로고    scopus 로고
    • High-effi ciency transduction of fi broblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy
    • Li M, Jayandharan GR, Li B, Ling C, Ma W, Srivastava A et al (2010) High-effi ciency transduction of fi broblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy. Hum Gene Ther 21:1527–1543
    • (2010) Hum Gene Ther , vol.21 , pp. 1527-1543
    • Li, M.1    Jayandharan, G.R.2    Li, B.3    Ling, C.4    Ma, W.5    Srivastava, A.6
  • 114
    • 54549096772 scopus 로고    scopus 로고
    • Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular traffi cking and transgene expression
    • Zhong L, Li B, Jayandharan G, Mah CS, Govindasamy L, Agbandje-McKenna M et al (2008) Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular traffi cking and transgene expression. Virology 381:194–202
    • (2008) Virology , vol.381 , pp. 194-202
    • Zhong, L.1    Li, B.2    Jayandharan, G.3    Mah, C.S.4    Govindasamy, L.5    Agbandje-McKenna, M.6
  • 115
    • 61649095399 scopus 로고    scopus 로고
    • Higheffi ciency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
    • Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ et al (2009) Higheffi ciency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 17:463–471
    • (2009) Mol Ther , vol.17 , pp. 463-471
    • Petrs-Silva, H.1    Dinculescu, A.2    Li, Q.3    Min, S.H.4    Chiodo, V.5    Pang, J.J.6
  • 117
    • 84878689856 scopus 로고    scopus 로고
    • Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice
    • Iida A, Takino N, Miyauchi H, Shimazaki K, Muramatsu S (2013) Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice. Biomed Res Int 2013:974819
    • (2013) Biomed Res Int , pp. 2013
    • Iida, A.1    Takino, N.2    Miyauchi, H.3    Shimazaki, K.4    Muramatsu, S.5
  • 119
    • 84878678218 scopus 로고    scopus 로고
    • Bioengineering of AAV2 capsid at specifi c serine, threonine, or lysine residues improves its transduction effi ciency in vitro and in vivo
    • Gabriel N, Hareendran S, Sen D, Gadkari RA, Sudha G, Selot R et al (2013) Bioengineering of AAV2 capsid at specifi c serine, threonine, or lysine residues improves its transduction effi ciency in vitro and in vivo. Hum Gene Ther Methods 24:80–93
    • (2013) Hum Gene Ther Methods , vol.24 , pp. 80-93
    • Gabriel, N.1    Hareendran, S.2    Sen, D.3    Gadkari, R.A.4    Sudha, G.5    Selot, R.6
  • 120
    • 67349133131 scopus 로고    scopus 로고
    • Preferential labeling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors
    • Nathanson JL, Yanagawa Y, Obata K, Callaway EM (2009) Preferential labeling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors. Neuroscience 161:441–450
    • (2009) Neuroscience , vol.161 , pp. 441-450
    • Nathanson, J.L.1    Yanagawa, Y.2    Obata, K.3    Callaway, E.M.4
  • 121
    • 17944378442 scopus 로고    scopus 로고
    • Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specifi c gene cassettes
    • Xu R, Janson CG, Mastakov M, Lawlor P, Young D, Mouravlev A et al (2001) Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specifi c gene cassettes. Gene Ther 8:1323–1332
    • (2001) Gene Ther , vol.8 , pp. 1323-1332
    • Xu, R.1    Janson, C.G.2    Mastakov, M.3    Lawlor, P.4    Young, D.5    Mouravlev, A.6
  • 122
    • 17144444172 scopus 로고    scopus 로고
    • Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units
    • Kugler S, Lingor P, Scholl U, Zolotukhin S, Bahr M (2003) Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units. Virology 311:89–95
    • (2003) Virology , vol.311 , pp. 89-95
    • Kugler, S.1    Lingor, P.2    Scholl, U.3    Zolotukhin, S.4    Bahr, M.5
  • 123
    • 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
    • Gray SJ, Foti SB, Schwartz JW, Bachaboina L, Taylor-Blake B, Coleman J et al (2011) Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum Gene Ther 22:1143–1153
    • (2011) Hum Gene Ther , vol.22 , pp. 1143-1153
    • Gray, S.J.1    Foti, S.B.2    Schwartz, J.W.3    Bachaboina, L.4    Taylor-Blake, B.5    Coleman, J.6
  • 124
    • 67749119683 scopus 로고    scopus 로고
    • Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters
    • Husain T, Passini MA, Parente MK, Fraser NW, Wolfe JH (2009) Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters. Gene Ther 16:927–932
    • (2009) Gene Ther , vol.16 , pp. 927-932
    • Husain, T.1    Passini, M.A.2    Parente, M.K.3    Fraser, N.W.4    Wolfe, J.H.5
  • 126
    • 0033870226 scopus 로고    scopus 로고
    • Infl uence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain
    • Paterna JC, Moccetti T, Mura A, Feldon J, Bueler H (2000) Infl uence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain. Gene Ther 7:1304–1311
    • (2000) Gene Ther , vol.7 , pp. 1304-1311
    • Paterna, J.C.1    Moccetti, T.2    Mura, A.3    Feldon, J.4    Bueler, H.5
  • 127
    • 11844288284 scopus 로고    scopus 로고
    • Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE)
    • Kingsman SM, Mitrophanous K, Olsen JC (2005) Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther 12:3–4
    • (2005) Gene Ther , vol.12 , pp. 3-4
    • Kingsman, S.M.1    Mitrophanous, K.2    Olsen, J.C.3
  • 128
    • 33645103149 scopus 로고    scopus 로고
    • Woodchuck hepatitis virus post- transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression
    • Schambach A, Bohne J, Baum C, Hermann FG, Egerer L, von Laer D et al (2006) Woodchuck hepatitis virus post- transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther 13:641–645
    • (2006) Gene Ther , vol.13 , pp. 641-645
    • Schambach, A.1    Bohne, J.2    Baum, C.3    Hermann, F.G.4    Egerer, L.5    Von Laer, D.6
  • 129
    • 79951510234 scopus 로고    scopus 로고
    • Vetter R et al (2011) microRNA122-regulated transgene expression increases specifi city of cardiac gene transfer upon intravenous delivery of AAV9 vectors
    • Geisler A, Jungmann A, Kurreck J, Poller W, Katus HA, Vetter R et al (2011) microRNA122-regulated transgene expression increases specifi city of cardiac gene transfer upon intravenous delivery of AAV9 vectors. Gene Ther 18:199–209
    • Gene Ther , vol.18 , pp. 199-209
    • Geisler, A.1    Jungmann, A.2    Kurreck, J.3    Poller, W.4    Katus, H.A.5
  • 131
    • 79953803239 scopus 로고    scopus 로고
    • Liver-specifi c microRNA-122 target sequences incorporated in AAV vectors effi ciently inhibits transgene expression in the liver
    • Qiao C, Yuan Z, Li J, He B, Zheng H, Mayer C et al (2011) Liver-specifi c microRNA-122 target sequences incorporated in AAV vectors effi ciently inhibits transgene expression in the liver. Gene Ther 18:403–410
    • (2011) Gene Ther , vol.18 , pp. 403-410
    • Qiao, C.1    Yuan, Z.2    Li, J.3    He, B.4    Zheng, H.5    Mayer, C.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.