-
1
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA 2008; 105: 15112-15117.
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 15112-15117
-
-
Cideciyan, A.V.1
Aleman, T.S.2
Boye, S.L.3
Schwartz, S.B.4
Kaushal, S.5
Roman, A.J.6
-
2
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
DOI 10.1056/NEJMoa0802315
-
Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008; 358: 2240-2248. (Pubitemid 351724453)
-
(2008)
New England Journal of Medicine
, vol.358
, Issue.21
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh Jr., E.N.4
Mingozzi, F.5
Bennicelli, J.6
Banfi, S.7
Marshall, K.A.8
Testa, F.9
Surace, E.M.10
Rossi, S.11
Lyubarsky, A.12
Arruda, V.R.13
Konkle, B.14
Stone, E.15
Sun, J.16
Jacobs, J.17
Dell'Osso, L.18
Hertle, R.19
Ma, J.-X.20
Redmond, T.M.21
Zhu, X.22
Hauck, B.23
Zelenaia, O.24
Shindler, K.S.25
Maguire, M.G.26
Wright, J.F.27
Volpe, N.J.28
McDonnell, J.W.29
Auricchio, A.30
High, K.A.31
Bennett, J.32
more..
-
3
-
-
50049136340
-
Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising
-
Bainbridge JW, Ali RR. Success in sight: the eyes have it! Ocular gene therapy trials for LCA look promising. Gene Therapy 2008; 15: 1191-1192.
-
(2008)
Gene Therapy
, vol.15
, pp. 1191-1192
-
-
Bainbridge, J.W.1
Ali, R.R.2
-
4
-
-
77952962177
-
Rod-derived cone viability factor for treating blinding diseases: From clinic to redox signaling
-
Léveillard T, Sahel J-A. Rod-derived cone viability factor for treating blinding diseases: from clinic to redox signaling. Science Trans Med 2010; 2: 26ps16.
-
(2010)
Science Trans Med
, vol.2
-
-
Léveillard, T.1
Sahel, J.-A.2
-
5
-
-
85049056931
-
Long-term retinal function and structure rescue using capsid mutant AAV8 Vector in the rd10 mouse, a model of recessive retinitis pigmentosa
-
Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S et al. Long-term retinal function and structure rescue using capsid mutant AAV8 Vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Mol Ther 2009; 19: 232-242.
-
(2009)
Mol Ther
, vol.19
, pp. 232-242
-
-
Pang, J.J.1
Dai, X.2
Boye, S.E.3
Barone, I.4
Boye, S.L.5
Mao, S.6
-
6
-
-
0035664438
-
Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa
-
DOI 10.1006/mthe.2001.0498
-
Sanftner L. Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol Ther 2001; 4: 622-629. (Pubitemid 34026878)
-
(2001)
Molecular Therapy
, vol.4
, Issue.6
, pp. 622-629
-
-
Sanftner, L.H.M.1
Abel, H.2
Hauswirth, W.W.3
Flannery, J.G.4
-
7
-
-
73849132937
-
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous
-
Dalkara D, Kolstad KD, Caporale N, Visel M, Klimczak RR, Schaffer DV et al. Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous. Mol Ther 2009; 17: 2096-2102.
-
(2009)
Mol Ther
, vol.17
, pp. 2096-2102
-
-
Dalkara, D.1
Kolstad, K.D.2
Caporale, N.3
Visel, M.4
Klimczak, R.R.5
Schaffer, D.V.6
-
8
-
-
77951960345
-
Changes in adeno-associated virus-mediated gene delivery in retinal degeneration
-
Kolstad KD, Dalkara D, Guerin K, Visel M, Hoffmann N, Schaffer DV et al. Changes in adeno-associated virus-mediated gene delivery in retinal degeneration. Hum Gene Ther 2010; 21: 571-578.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 571-578
-
-
Kolstad, K.D.1
Dalkara, D.2
Guerin, K.3
Visel, M.4
Hoffmann, N.5
Schaffer, D.V.6
-
9
-
-
79551634862
-
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina
-
Petrs-Silva H, Dinculescu A, Li Q, Deng W-T, Pang J-j, Min S-H et al. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther 2009; 19: 293-301.
-
(2009)
Mol Ther
, vol.19
, pp. 293-301
-
-
Petrs-Silva, H.1
Dinculescu, A.2
Li, Q.3
Deng, W.-T.4
J-J, P.5
Min, S.-H.6
-
10
-
-
61649095399
-
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
-
Petrs-Silva H, Dinculescu A, Li Q, Min S-H, Chiodo V, Pang J-j et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 2009; 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
J-J, P.6
-
11
-
-
70449338146
-
A novel adenoassociated viral variant for efficient and selective intravitreal transduction of rat Müller cells
-
Klimczak RR, Koerber JT, Dalkara D, Flannery JG, Schaffer DV. A novel adenoassociated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PloS One 2009; 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
-
12
-
-
73849091644
-
Molecular evolution of adeno-associated virus for enhanced glial gene delivery
-
Koerber JT, Klimczak R, Jang J-H, Dalkara D, Flannery JG, Schaffer DV. 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
Dalkara, D.4
Flannery, J.G.5
Schaffer, D.V.6
-
13
-
-
79956006692
-
Intravitreal injection of AAV2 transduces macaque inner retina
-
Yin L, Greenberg K, Hunter JJ, Dalkara D, Kolstad KD, Masella BD et al. Intravitreal injection of AAV2 transduces macaque inner retina. IOVS 2011; 52: 2775-2783.
-
(2011)
IOVS
, vol.52
, pp. 2775-2783
-
-
Yin, L.1
Greenberg, K.2
Hunter, J.J.3
Dalkara, D.4
Kolstad, K.D.5
Masella, B.D.6
-
14
-
-
0034870041
-
The fundamental plan of the retina
-
DOI 10.1038/nn0901-877
-
Masland RH. The fundamental plan of the retina. Nat Neurosci 2001; 4: 877-886. (Pubitemid 32801590)
-
(2001)
Nature Neuroscience
, vol.4
, Issue.9
, pp. 877-886
-
-
Masland, R.H.1
-
15
-
-
79959932534
-
Immune responses to AAV in clinical trials
-
Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther 2011; 11: 321-330.
-
(2011)
Curr Gene Ther
, vol.11
, pp. 321-330
-
-
Mingozzi, F.1
High, K.A.2
-
16
-
-
35848961457
-
Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration
-
DOI 10.1038/sj.gt.3303029, PII 3303029
-
Bostick B, Ghosh A, Yue Y, Long C, Duan D. Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Therapy 2007; 14: 1605-1609. (Pubitemid 350056232)
-
(2007)
Gene Therapy
, vol.14
, Issue.22
, pp. 1605-1609
-
-
Bostick, B.1
Ghosh, A.2
Yue, Y.3
Long, C.4
Duan, D.5
-
17
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2008; 27: 59-65.
-
(2008)
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
-
18
-
-
77749249680
-
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
-
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM 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
Braun, L.4
Bevan, A.K.5
Haidet, A.M.6
-
19
-
-
79961032418
-
Several rAAV vectors efficiently 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. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 2009; 19: 1440-1448.
-
(2009)
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
-
20
-
-
80053923197
-
Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
-
Rahim AA, Wong AM, Hoefer K, Buckley SM, Mattar CN, Cheng SH et al. Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J 2011; 25: 3505-3518.
-
(2011)
FASEB J
, vol.25
, pp. 3505-3518
-
-
Rahim, A.A.1
Wong, A.M.2
Hoefer, K.3
Buckley, S.M.4
Mattar, C.N.5
Cheng, S.H.6
-
21
-
-
33746874152
-
Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy
-
DOI 10.1016/j.ymthe.2006.05.009, PII S1525001606002048
-
Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006; 14: 316-327. (Pubitemid 44184972)
-
(2006)
Molecular Therapy
, vol.14
, Issue.3
, pp. 316-327
-
-
Wu, Z.1
Asokan, A.2
Samulski, R.J.3
-
22
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
-
DOI 10.1073/pnas.0802866105
-
Zhong L, Li B, Mah CS, Govindasamy L, Agbandje-McKenna M, Cooper M et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci USA 2008; 105: 7827-7832. (Pubitemid 351872723)
-
(2008)
Proceedings of the National Academy of Sciences of the United States of America
, vol.105
, Issue.22
, pp. 7827-7832
-
-
Zhong, L.1
Li, B.2
Mah, C.S.3
Govindasamy, L.4
Agbandje-McKenna, M.5
Cooper, M.6
Herzog, R.W.7
Zolotukhin, I.8
Warrington Jr., K.H.9
Weigel-Van Aken, K.A.10
Hobbs, J.A.11
Zolotukhin, S.12
Muzyczka, N.13
Srivastava, A.14
-
23
-
-
16844379844
-
Viral vector mediated overexpression of human α-synuclein in the nigrostriatal dopaminergic neurons: A new model for Parkinson's disease
-
Maingay M, Romero-Ramos M. Viral vector mediated overexpression of human alphasynuclein in the nigrostriatal dopaminergic neurons: a new model for Parkinson's disease. CNS Spectr 2005; 10: 235-244. (Pubitemid 40489292)
-
(2005)
CNS Spectrums
, vol.10
, Issue.3
, pp. 235-244
-
-
Maingay, M.1
Romero-Ramos, M.2
Kirik, D.3
-
24
-
-
77957224859
-
Viral vector-mediated overexpression of alpha-synuclein as a progressive model of Parkinson's disease
-
Ulusoy A, Decressac M, Kirik D, Bjorklund A. Viral vector-mediated overexpression of alpha-synuclein as a progressive model of Parkinson's disease. Prog Brain Res 2010; 184: 89-111.
-
(2010)
Prog Brain Res
, vol.184
, pp. 89-111
-
-
Ulusoy, A.1
Decressac, M.2
Kirik, D.3
Bjorklund, A.4
-
25
-
-
67650218487
-
MicroRNAs-powerful repression comes from small RNAs
-
Ma C, Liu Y, He L. MicroRNAs-powerful repression comes from small RNAs. Science China C Life Sci 2009; 52: 323-330.
-
(2009)
Science China C Life Sci
, vol.52
, pp. 323-330
-
-
Ma, C.1
Liu, Y.2
He, L.3
-
26
-
-
34347252689
-
Production and characterization of adeno-associated viral vectors
-
Grieger JC, Choi VW, Samulski RJ. Production and characterization of adeno-associated viral vectors. Nat Protoc 2006; 1: 1412-1428.
-
(2006)
Nat Protoc
, vol.1
, pp. 1412-1428
-
-
Grieger, J.C.1
Choi, V.W.2
Samulski, R.J.3
|