| 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 | 1247 | 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 | 848 | 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 | 829 | 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 | 785 | 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 | 430 | 2009 |
| A comprehensive review of retinal gene therapy SE Boye, SL Boye, AS Lewin, WW Hauswirth Molecular therapy 21 (3), 509-519, 2013 | 365 | 2013 |
| 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 | 364 | 2011 |
| 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 | 346 | 2012 |
| 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 (3), 327-343, 2016 | 302 | 2016 |
| 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 | 279 | 2013 |
| 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 | 279 | 2009 |
| 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 | 272 | 2006 |
| 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 | 214 | 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 | 210 | 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 | 189 | 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 | 186 | 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 | 179 | 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 | 170 | 2008 |
| Dual Adeno-Associated Virus Vectors Result in Efficient In Vitro and In Vivo Expression of an Oversized Gene, MYO7A FM Dyka, SL Boye, VA Chiodo, WW Hauswirth, SE Boye Human gene therapy methods 25 (2), 166-177, 2014 | 151 | 2014 |
| 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 | 149 | 2012 |
william hauswirthuniversity of floridaVerified email at eye.ufl.edu
