| Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial WW Hauswirth, TS Aleman, S Kaushal, AV Cideciyan, SB Schwartz, ... Human gene therapy 19 (10), 979-990, 2008 | 1197 | 2008 |
| Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics AV Cideciyan, TS Aleman, SL Boye, SB Schwartz, S Kaushal, AJ Roman, ... Proceedings of the National Academy of Sciences 105 (39), 15112-15117, 2008 | 823 | 2008 |
| Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years SG Jacobson, AV Cideciyan, R Ratnakaram, E Heon, SB Schwartz, ... Archives of ophthalmology 130 (1), 9-24, 2012 | 778 | 2012 |
| Current clinical applications of in vivo gene therapy with AAVs JR Mendell, SA Al-Zaidy, LR Rodino-Klapac, K Goodspeed, SJ Gray, ... Molecular Therapy 29 (2), 464-488, 2021 | 551 | 2021 |
| Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year AV Cideciyan, WW Hauswirth, TS Aleman, S Kaushal, SB Schwartz, ... Human gene therapy 20 (9), 999-1004, 2009 | 427 | 2009 |
| Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness MM Doroudchi, KP Greenberg, J Liu, KA Silka, ES Boyden, JA Lockridge, ... Molecular Therapy 19 (7), 1220-1229, 2011 | 347 | 2011 |
| A comprehensive review of retinal gene therapy SE Boye, SL Boye, AS Lewin, WW Hauswirth Molecular therapy 21 (3), 509-519, 2013 | 345 | 2013 |
| Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa WA Beltran, AV Cideciyan, AS Lewin, S Iwabe, H Khanna, A Sumaroka, ... Proceedings of the National Academy of Sciences 109 (6), 2132-2137, 2012 | 321 | 2012 |
| Vision 1 year after gene therapy for Leber's congenital amaurosis AV Cideciyan, WW Hauswirth, TS Aleman, S Kaushal, SB Schwartz, ... New England Journal of Medicine 361 (7), 725-727, 2009 | 271 | 2009 |
| Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial NG Ghazi, EB Abboud, SR Nowilaty, H Alkuraya, A Alhommadi, H Cai, ... Human genetics 135, 327-343, 2016 | 267 | 2016 |
| Safety of recombinant adeno-associated virus type 2–RPE65 vector delivered by ocular subretinal injection SG Jacobson, GM Acland, GD Aguirre, TS Aleman, SB Schwartz, ... Molecular Therapy 13 (6), 1074-1084, 2006 | 256 | 2006 |
| Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death DS Welsbie, Z Yang, Y Ge, KL Mitchell, X Zhou, SE Martin, CA Berlinicke, ... Proceedings of the National Academy of Sciences 110 (10), 4045-4050, 2013 | 248 | 2013 |
| Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors CN Kay, RC Ryals, GV Aslanidi, SH Min, Q Ruan, J Sun, FM Dyka, ... PloS one 8 (4), e62097, 2013 | 202 | 2013 |
| Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model H Yu, RD Koilkonda, TH Chou, V Porciatti, SS Ozdemir, V Chiodo, ... Proceedings of the National Academy of Sciences 109 (20), E1238-E1247, 2012 | 187 | 2012 |
| Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa J Pang, X Dai, SE Boye, I Barone, SL Boye, S Mao, D Everhart, ... Molecular therapy 19 (2), 234-242, 2011 | 183 | 2011 |
| Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis SG Jacobson, SL Boye, TS Aleman, TJ Conlon, CJ Zeiss, AJ Roman, ... Human gene therapy 17 (8), 845-858, 2006 | 171 | 2006 |
| Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus VS Lopes, SE Boye, CM Louie, S Boye, F Dyka, V Chiodo, H Fofo, ... Gene Therapy, 2013 | 163 | 2013 |
| AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEβ mutation J Pang, SL Boye, A Kumar, A Dinculescu, W Deng, J Li, Q Li, A Rani, ... Investigative ophthalmology & visual science 49 (10), 4278-4283, 2008 | 163 | 2008 |
| AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia J Pang, WT Deng, X Dai, B Lei, D Everhart, Y Umino, J Li, K Zhang, S Mao, ... PloS one 7 (4), e35250, 2012 | 137 | 2012 |
| The human rhodopsin kinase promoter in an AAV5 vector confers rod-and cone-specific expression in the primate retina SE Boye, JJ Alexander, SL Boye, CD Witherspoon, KJ Sandefer, ... Human gene therapy 23 (10), 1101-1115, 2012 | 136 | 2012 |
william hauswirthuniversity of floridaVerified email at eye.ufl.edu
