-
1
-
-
84971264666
-
Evaluation of gene therapy as an intervention strategy to treat brain injury from stroke
-
1 Craig, AJ, Housley, GD, Evaluation of gene therapy as an intervention strategy to treat brain injury from stroke. Front Mol Neurosci, 9, 2016, 34.
-
(2016)
Front Mol Neurosci
, vol.9
, pp. 34
-
-
Craig, A.J.1
Housley, G.D.2
-
2
-
-
84948448009
-
Gene therapy for the treatment of neurological disorders: metabolic disorders
-
2 Gessler, DJ, Gao, G, Gene therapy for the treatment of neurological disorders: metabolic disorders. Methods Mol Biol 1382 (2016), 429–465.
-
(2016)
Methods Mol Biol
, vol.1382
, pp. 429-465
-
-
Gessler, D.J.1
Gao, G.2
-
3
-
-
84901832947
-
Systemic gene delivery to the central nervous system using adeno-associated virus
-
3 Bourdenx, M, Dutheil, N, Bezard, E, Dehay, B, Systemic gene delivery to the central nervous system using adeno-associated virus. Front Mol Neurosci, 7, 2014, 50.
-
(2014)
Front Mol Neurosci
, vol.7
, pp. 50
-
-
Bourdenx, M.1
Dutheil, N.2
Bezard, E.3
Dehay, B.4
-
4
-
-
84885164755
-
Gene therapy for psychiatric disorders
-
4 Gelfand, Y, Kaplitt, MG, Gene therapy for psychiatric disorders. World Neurosurg 80 (2013), S32.e11–S32.e18.
-
(2013)
World Neurosurg
, vol.80
, pp. S32.e11-S32.e18
-
-
Gelfand, Y.1
Kaplitt, M.G.2
-
5
-
-
85007302654
-
AAV2 gene therapy readministration in three adults with congenital blindness
-
5 Bennett, J, Ashtari, M, Wellman, J, Marshall, KA, Cyckowski, LL, Chung, DC, et al. AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med, 4, 2012, 120ra15.
-
(2012)
Sci Transl Med
, vol.4
, pp. 120ra15
-
-
Bennett, J.1
Ashtari, M.2
Wellman, J.3
Marshall, K.A.4
Cyckowski, L.L.5
Chung, D.C.6
-
6
-
-
70350620424
-
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
-
6 Maguire, AM, High, KA, Auricchio, A, Wright, JF, Pierce, EA, Testa, F, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374 (2009), 1597–1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
Wright, J.F.4
Pierce, E.A.5
Testa, F.6
-
7
-
-
44349136847
-
Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
-
7 Worgall, S, Sondhi, D, Hackett, NR, Kosofsky, B, Kekatpure, MV, Neyzi, N, 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 19 (2008), 463–474.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 463-474
-
-
Worgall, S.1
Sondhi, D.2
Hackett, N.R.3
Kosofsky, B.4
Kekatpure, M.V.5
Neyzi, N.6
-
8
-
-
0037143294
-
Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain
-
8 Janson, C, McPhee, S, Bilaniuk, L, Haselgrove, J, Testaiuti, M, Freese, A, et al. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther 13 (2002), 1391–1412.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1391-1412
-
-
Janson, C.1
McPhee, S.2
Bilaniuk, L.3
Haselgrove, J.4
Testaiuti, M.5
Freese, A.6
-
9
-
-
84930532618
-
A single injection of recombinant adeno-associated virus into the lumbar cistern delivers transgene expression throughout the whole spinal cord
-
9 Guo, Y, Wang, D, Qiao, T, Yang, C, Su, Q, Gao, G, et al. A single injection of recombinant adeno-associated virus into the lumbar cistern delivers transgene expression throughout the whole spinal cord. Mol Neurobiol 53 (2016), 3235–3248.
-
(2016)
Mol Neurobiol
, vol.53
, pp. 3235-3248
-
-
Guo, Y.1
Wang, D.2
Qiao, T.3
Yang, C.4
Su, Q.5
Gao, G.6
-
10
-
-
84939523769
-
Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector
-
10 Hironaka, K, Yamazaki, Y, Hirai, Y, Yamamoto, M, Miyake, N, Miyake, K, et al. Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector. Sci Rep, 5, 2015, 13104.
-
(2015)
Sci Rep
, vol.5
, pp. 13104
-
-
Hironaka, K.1
Yamazaki, Y.2
Hirai, Y.3
Yamamoto, M.4
Miyake, N.5
Miyake, K.6
-
11
-
-
84926421140
-
Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
-
11 Hordeaux, J, Dubreil, L, Deniaud, J, Iacobelli, F, Moreau, S, Ledevin, M, et al. Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats. Gene Ther 22 (2015), 316–324.
-
(2015)
Gene Ther
, vol.22
, pp. 316-324
-
-
Hordeaux, J.1
Dubreil, L.2
Deniaud, J.3
Iacobelli, F.4
Moreau, S.5
Ledevin, M.6
-
12
-
-
84927155708
-
Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I
-
12 Hinderer, C, Bell, P, Gurda, BL, Wang, Q, Louboutin, JP, Zhu, Y, et al. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I. Mol Ther 22 (2014), 2018–2027.
-
(2014)
Mol Ther
, vol.22
, pp. 2018-2027
-
-
Hinderer, C.1
Bell, P.2
Gurda, B.L.3
Wang, Q.4
Louboutin, J.P.5
Zhu, Y.6
-
13
-
-
84881228888
-
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
-
13 Haurigot, V, Marcó, S, Ribera, A, Garcia, M, Ruzo, A, Villacampa, P, et al. Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J Clin Invest 123:8 (2013), 3254–3271.
-
(2013)
J Clin Invest
, vol.123
, Issue.8
, pp. 3254-3271
-
-
Haurigot, V.1
Marcó, S.2
Ribera, A.3
Garcia, M.4
Ruzo, A.5
Villacampa, P.6
-
14
-
-
84890365334
-
Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain
-
14 Hudry, E, Dashkoff, J, Roe, AD, Takeda, S, Koffie, RM, Hashimoto, T, et al. Gene transfer of human Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain. Sci Transl Med, 5, 2013, 212ra161.
-
(2013)
Sci Transl Med
, vol.5
, pp. 212ra161
-
-
Hudry, E.1
Dashkoff, J.2
Roe, A.D.3
Takeda, S.4
Koffie, R.M.5
Hashimoto, T.6
-
15
-
-
33751111439
-
Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice
-
15 Levites, Y, Jansen, K, Smithson, LA, Dakin, R, Holloway, VM, Das, P, et al. Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice. J Neurosci 26 (2006), 11923–11928.
-
(2006)
J Neurosci
, vol.26
, pp. 11923-11928
-
-
Levites, Y.1
Jansen, K.2
Smithson, L.A.3
Dakin, R.4
Holloway, V.M.5
Das, P.6
-
16
-
-
27144545459
-
Intrathecal gene transfer by adeno-associated virus for pain
-
16 Beutler, AS, Banck, MS, Walsh, CE, Milligan, ED, Intrathecal gene transfer by adeno-associated virus for pain. Curr Opin Mol Ther 7 (2005), 431–439.
-
(2005)
Curr Opin Mol Ther
, vol.7
, pp. 431-439
-
-
Beutler, A.S.1
Banck, M.S.2
Walsh, C.E.3
Milligan, E.D.4
-
17
-
-
84902676801
-
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
-
17 Murrey, DA, Naughton, BJ, Duncan, FJ, Meadows, AS, Ware, TA, Campbell, KJ, 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 25 (2014), 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
Meadows, A.S.4
Ware, T.A.5
Campbell, K.J.6
-
18
-
-
84871994994
-
Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems
-
18 Mattar, CN, Waddington, SN, Biswas, A, Johana, N, Ng, XW, Fisk, AS, et al. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Gene Ther 20 (2013), 69–83.
-
(2013)
Gene Ther
, vol.20
, pp. 69-83
-
-
Mattar, C.N.1
Waddington, S.N.2
Biswas, A.3
Johana, N.4
Ng, X.W.5
Fisk, A.S.6
-
19
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
19 Foust, KD, Nurre, E, Montgomery, CL, Hernandez, A, Chan, CM, Kaspar, BK, Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27 (2009), 59–65.
-
(2009)
Nat Biotechnol
, vol.27
, pp. 59-65
-
-
Foust, K.D.1
Nurre, E.2
Montgomery, C.L.3
Hernandez, A.4
Chan, C.M.5
Kaspar, B.K.6
-
20
-
-
79960696509
-
Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model
-
20 Pleger, ST, Shan, C, Ksienzyk, J, Bekeredjian, R, Boekstegers, P, Hinkel, R, et al. Cardiac AAV9-S100A1 gene therapy rescues post-ischemic heart failure in a preclinical large animal model. Sci Transl Med, 3, 2011, 92ra64.
-
(2011)
Sci Transl Med
, vol.3
, pp. 92ra64
-
-
Pleger, S.T.1
Shan, C.2
Ksienzyk, J.3
Bekeredjian, R.4
Boekstegers, P.5
Hinkel, R.6
-
21
-
-
84959091629
-
Exosome-associated AAV vector as a robust and convenient neuroscience tool
-
21 Hudry, E, Martin, C, Gandhi, S, György, B, Scheffer, DI, Mu, D, et al. Exosome-associated AAV vector as a robust and convenient neuroscience tool. Gene Ther 23 (2016), 380–392.
-
(2016)
Gene Ther
, vol.23
, pp. 380-392
-
-
Hudry, E.1
Martin, C.2
Gandhi, S.3
György, B.4
Scheffer, D.I.5
Mu, D.6
-
22
-
-
84957588611
-
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
-
22 Deverman, BE, Pravdo, PL, Simpson, BP, Kumar, SR, Chan, KY, Banerjee, A, et al. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat Biotechnol 34 (2016), 204–209.
-
(2016)
Nat Biotechnol
, vol.34
, pp. 204-209
-
-
Deverman, B.E.1
Pravdo, P.L.2
Simpson, B.P.3
Kumar, S.R.4
Chan, K.Y.5
Banerjee, A.6
-
23
-
-
79957888016
-
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates
-
23 Gray, SJ, Matagne, V, Bachaboina, L, Yadav, S, Ojeda, SR, Samulski, RJ, Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol Ther 19 (2011), 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
-
24
-
-
54849162100
-
Self-complementary AAV vectors; advances and applications
-
24 McCarty, DM, Self-complementary AAV vectors; advances and applications. Mol Ther 16 (2008), 1648–1656.
-
(2008)
Mol Ther
, vol.16
, pp. 1648-1656
-
-
McCarty, D.M.1
-
25
-
-
84875866006
-
Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice
-
25 Iwata, N, Sekiguchi, M, Hattori, Y, Takahashi, A, Asai, M, Ji, B, et al. Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice. Sci Rep, 3, 2013, 1472.
-
(2013)
Sci Rep
, vol.3
, pp. 1472
-
-
Iwata, N.1
Sekiguchi, M.2
Hattori, Y.3
Takahashi, A.4
Asai, M.5
Ji, B.6
-
26
-
-
79957882063
-
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
-
26 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 19 (2011), 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
-
27
-
-
84873410199
-
Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice
-
27 Benkhelifa-Ziyyat, S, Besse, A, Roda, M, Duque, S, Astord, S, Carcenac, R, et al. Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther 21 (2013), 282–290.
-
(2013)
Mol Ther
, vol.21
, pp. 282-290
-
-
Benkhelifa-Ziyyat, S.1
Besse, A.2
Roda, M.3
Duque, S.4
Astord, S.5
Carcenac, R.6
-
28
-
-
84887084505
-
Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons
-
28 Yamashita, T, Chai, HL, Teramoto, S, Tsuji, S, Shimazaki, K, Muramatsu, S, et al. Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons. EMBO Mol Med 5 (2013), 1710–1719.
-
(2013)
EMBO Mol Med
, vol.5
, pp. 1710-1719
-
-
Yamashita, T.1
Chai, H.L.2
Teramoto, S.3
Tsuji, S.4
Shimazaki, K.5
Muramatsu, S.6
-
29
-
-
0034749458
-
Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors
-
29 Sarukhan, A, Camugli, S, Gjata, B, von Boehmer, H, Danos, O, Jooss, K, Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors. J Virol 75 (2001), 269–277.
-
(2001)
J Virol
, vol.75
, pp. 269-277
-
-
Sarukhan, A.1
Camugli, S.2
Gjata, B.3
von Boehmer, H.4
Danos, O.5
Jooss, K.6
-
30
-
-
0032977833
-
Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration
-
30 Brockstedt, DG, Podsakoff, GM, Fong, L, Kurtzman, G, Mueller-Ruchholtz, W, Engleman, EG, Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration. Clin Immunol 92 (1999), 67–75.
-
(1999)
Clin Immunol
, vol.92
, pp. 67-75
-
-
Brockstedt, D.G.1
Podsakoff, G.M.2
Fong, L.3
Kurtzman, G.4
Mueller-Ruchholtz, W.5
Engleman, E.G.6
-
31
-
-
44349170706
-
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
-
31 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 16 (2008), 1073–1080.
-
(2008)
Mol Ther
, vol.16
, pp. 1073-1080
-
-
Zincarelli, C.1
Soltys, S.2
Rengo, G.3
Rabinowitz, J.E.4
-
32
-
-
84932614435
-
Distinct transduction profiles in the CNS via three injection routes of AAV9 and the application to generation of a neurodegenerative mouse model
-
32 Huda, F, Konno, A, Matsuzaki, Y, Goenawan, H, Miyake, K, Shimada, T, et al. Distinct transduction profiles in the CNS via three injection routes of AAV9 and the application to generation of a neurodegenerative mouse model. Mol Ther Methods Clin Dev, 1, 2014, 14032.
-
(2014)
Mol Ther Methods Clin Dev
, vol.1
, pp. 14032
-
-
Huda, F.1
Konno, A.2
Matsuzaki, Y.3
Goenawan, H.4
Miyake, K.5
Shimada, T.6
-
33
-
-
70349850388
-
Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
-
33 Lawlor, PA, Bland, RJ, Mouravlev, A, Young, D, During, MJ, Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol Ther 17 (2009), 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
-
34
-
-
0037297303
-
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
-
34 Kügler, S, Kilic, E, Bähr, 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 10 (2003), 337–347.
-
(2003)
Gene Ther
, vol.10
, pp. 337-347
-
-
Kügler, S.1
Kilic, E.2
Bähr, M.3
-
35
-
-
44449173870
-
GFAP promoter elements required for region-specific and astrocyte-specific expression
-
35 Lee, Y, Messing, A, Su, M, Brenner, M, GFAP promoter elements required for region-specific and astrocyte-specific expression. Glia 56 (2008), 481–493.
-
(2008)
Glia
, vol.56
, pp. 481-493
-
-
Lee, Y.1
Messing, A.2
Su, M.3
Brenner, M.4
-
36
-
-
33947410764
-
Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity
-
36 Wu, J, Zhao, W, Zhong, L, Han, Z, Li, B, Ma, W, et al. Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity. Hum Gene Ther 18 (2007), 171–182.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 171-182
-
-
Wu, J.1
Zhao, W.2
Zhong, L.3
Han, Z.4
Li, B.5
Ma, W.6
-
37
-
-
84881293648
-
Mouse gender influences brain transduction by intravascularly administered AAV9
-
37 Maguire, CA, Crommentuijn, MH, Mu, D, Hudry, E, Serrano-Pozo, A, Hyman, BT, et al. Mouse gender influences brain transduction by intravascularly administered AAV9. Mol Ther 21 (2013), 1470–1471.
-
(2013)
Mol Ther
, vol.21
, pp. 1470-1471
-
-
Maguire, C.A.1
Crommentuijn, M.H.2
Mu, D.3
Hudry, E.4
Serrano-Pozo, A.5
Hyman, B.T.6
-
38
-
-
84900988576
-
Glutamine synthetase as an astrocytic marker: its cell type and vesicle localization
-
38 Anlauf, E, Derouiche, A, Glutamine synthetase as an astrocytic marker: its cell type and vesicle localization. Front Endocrinol (Lausanne), 4, 2013, 144.
-
(2013)
Front Endocrinol (Lausanne)
, vol.4
, pp. 144
-
-
Anlauf, E.1
Derouiche, A.2
-
39
-
-
84871905759
-
Attenuating astrocyte activation accelerates plaque pathogenesis in APP/PS1 mice
-
39 Kraft, AW, Hu, X, Yoon, H, Yan, P, Xiao, Q, Wang, Y, et al. Attenuating astrocyte activation accelerates plaque pathogenesis in APP/PS1 mice. FASEB J 27 (2013), 187–198.
-
(2013)
FASEB J
, vol.27
, pp. 187-198
-
-
Kraft, A.W.1
Hu, X.2
Yoon, H.3
Yan, P.4
Xiao, Q.5
Wang, Y.6
-
40
-
-
0348038748
-
Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
-
40 Wang, Z, Ma, HI, Li, J, Sun, L, Zhang, J, Xiao, X, Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther 10 (2003), 2105–2111.
-
(2003)
Gene Ther
, vol.10
, pp. 2105-2111
-
-
Wang, Z.1
Ma, H.I.2
Li, J.3
Sun, L.4
Zhang, J.5
Xiao, X.6
-
41
-
-
0034882826
-
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
-
41 McCarty, DM, Monahan, PE, Samulski, RJ, Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8 (2001), 1248–1254.
-
(2001)
Gene Ther
, vol.8
, pp. 1248-1254
-
-
McCarty, D.M.1
Monahan, P.E.2
Samulski, R.J.3
-
42
-
-
84905455206
-
Dual transgene expression in murine cerebellar Purkinje neurons by viral transduction in vivo
-
42 Bosch, MK, Nerbonne, JM, Ornitz, DM, Dual transgene expression in murine cerebellar Purkinje neurons by viral transduction in vivo. PLoS One, 9, 2014, e104062.
-
(2014)
PLoS One
, vol.9
, pp. e104062
-
-
Bosch, M.K.1
Nerbonne, J.M.2
Ornitz, D.M.3
-
43
-
-
84883268937
-
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
-
43 Mingozzi, F, High, KA, Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood 122 (2013), 23–36.
-
(2013)
Blood
, vol.122
, pp. 23-36
-
-
Mingozzi, F.1
High, K.A.2
-
44
-
-
21244441929
-
Immune responses to adeno-associated virus vectors
-
44 Zaiss, AK, Muruve, DA, Immune responses to adeno-associated virus vectors. Curr Gene Ther 5 (2005), 323–331.
-
(2005)
Curr Gene Ther
, vol.5
, pp. 323-331
-
-
Zaiss, A.K.1
Muruve, D.A.2
-
45
-
-
84907169653
-
Glia in the pathogenesis of neurodegenerative diseases
-
45 Verkhratsky, A, Parpura, V, Pekna, M, Pekny, M, Sofroniew, M, Glia in the pathogenesis of neurodegenerative diseases. Biochem Soc Trans 42 (2014), 1291–1301.
-
(2014)
Biochem Soc Trans
, vol.42
, pp. 1291-1301
-
-
Verkhratsky, A.1
Parpura, V.2
Pekna, M.3
Pekny, M.4
Sofroniew, M.5
-
46
-
-
33751237994
-
Mechanisms of disease: astrocytes in neurodegenerative disease
-
46 Maragakis, NJ, Rothstein, JD, Mechanisms of disease: astrocytes in neurodegenerative disease. Nat Clin Pract Neurol 2 (2006), 679–689.
-
(2006)
Nat Clin Pract Neurol
, vol.2
, pp. 679-689
-
-
Maragakis, N.J.1
Rothstein, J.D.2
-
47
-
-
84943303109
-
Distinct expression patterns of AAV8 vectors with broadly active promoters from subretinal injections of neonatal mouse eyes at two different ages
-
47 Xiong, W, Cepko, C, Distinct expression patterns of AAV8 vectors with broadly active promoters from subretinal injections of neonatal mouse eyes at two different ages. Adv Exp Med Biol 854 (2016), 501–507.
-
(2016)
Adv Exp Med Biol
, vol.854
, pp. 501-507
-
-
Xiong, W.1
Cepko, C.2
-
48
-
-
84955567365
-
Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector
-
48 Choudhury, SR, Harris, AF, Cabral, DJ, Keeler, AM, Sapp, E, Ferreira, JS, et al. Widespread central nervous system gene transfer and silencing after systemic delivery of novel AAV-AS vector. Mol Ther 24 (2016), 726–735.
-
(2016)
Mol Ther
, vol.24
, pp. 726-735
-
-
Choudhury, S.R.1
Harris, A.F.2
Cabral, D.J.3
Keeler, A.M.4
Sapp, E.5
Ferreira, J.S.6
-
49
-
-
84903760379
-
Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10
-
49 Yang, B, Li, S, Wang, H, Guo, Y, Gessler, DJ, Cao, C, et al. Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther 22 (2014), 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
-
50
-
-
0030833055
-
Accelerated amyloid deposition in the brains of transgenic mice coexpressing mutant presenilin 1 and amyloid precursor proteins
-
50 Borchelt, DR, Ratovitski, T, van Lare, J, Lee, MK, Gonzales, V, Jenkins, NA, et al. Accelerated amyloid deposition in the brains of transgenic mice coexpressing mutant presenilin 1 and amyloid precursor proteins. Neuron 19 (1997), 939–945.
-
(1997)
Neuron
, vol.19
, pp. 939-945
-
-
Borchelt, D.R.1
Ratovitski, T.2
van Lare, J.3
Lee, M.K.4
Gonzales, V.5
Jenkins, N.A.6
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