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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
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published online 22 January (doi: 10.1073/pnas.1218933110
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A. V. Cideciyan, S. G. Jacobson, W. A. Beltran, A. Sumaroka, M. Swider, S. Iwabe, A. J. Roman, M. B. Olivares, S. B. Schwartz, A. M. Komaromy, W. W. Hauswirth, G. D. Aguirre, Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc. Natl. Acad. Sci. U.S.A., published online 22 January 2013 (doi: 10.1073/pnas.1218933110).
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Cideciyan, A.V.1
Jacobson, S.G.2
Beltran, W.A.3
Sumaroka, A.4
Swider, M.5
Iwabe, S.6
Roman, A.J.7
Olivares, M.B.8
Schwartz, S.B.9
Komaromy, A.M.10
Hauswirth, W.W.11
Aguirre, G.D.12
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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
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G. M. Acland, G. D. Aguirre, J. Bennett, T. S. Aleman, A. V. Cideciyan, J. Bennicelli, N. S. Dejneka, S. E. Pearce-Kelling, A. M. Maguire, K. Palczewski, W. W. Hauswirth, S. G. Jacobson, 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). (Pubitemid 41723041)
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Gene therapy restores vision in a canine model of childhood blindness
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DOI 10.1038/88327
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G. M. Acland, G. D. Aguirre, J. Ray, Q. Zhang, T. S. Aleman, A. V. Cideciyan, S. E. Pearce-Kelling, V. Anand, Y. Zeng, A. M. Maguire, S. G. Jacobson, W. W. Hauswirth, J. Bennett, Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet. 28, 92-95 (2001). (Pubitemid 32405824)
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: Safety and effi cacy in 15 children and adults followed up to 3 years
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S. G. Jacobson, A. V. Cideciyan, R. Ratnakaram, E. Heon, S. B. Schwartz, A. J. Roman, M. C. Peden, T. S. Aleman, S. L. Boye, A. Sumaroka, T. J. Conlon, R. Calcedo, J. J. Pang, K. E. Erger, M. B. Olivares, C. L. Mullins, M. Swider, S. Kaushal, W. J. Feuer, A. Iannaccone, G. A. Fishman, E. M. Stone, B. J. Byrne, W. W. Hauswirth, Gene therapy for leber congenital amaurosis caused by RPE65 mutations: Safety and effi cacy in 15 children and adults followed up to 3 years. Arch. Ophthalmol. 130, 9-24 (2012).
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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Shortterm results of a phase 1 trial
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W. W. Hauswirth, T. S. Aleman, S. Kaushal, A. V. Cideciyan, S. B. Schwartz, L. Wang, T. J. Conlon, S. L. Boye, T. R. Flotte, B. J. Byrne, S. G. Jacobson, Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Shortterm results of a phase 1 trial. Hum. Gene Ther. 19, 979-990 (2008).
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Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa
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W. A. Beltran, A. V. Cideciyan, A. S. Lewin, S. Iwabe, H. Khanna, A. Sumaroka, V. A. Chiodo, D. S. Fajardo, A. J. Román, W. T. Deng, M. Swider, T. S. Alemán, S. L. Boye, S. Genini, A. Swaroop, W. W. Hauswirth, S. G. Jacobson, G. D. Aguirre, Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proc. Natl. Acad. Sci. U.S.A. 109, 2132-2137 (2012).
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Aguirre, G.D.18
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