| 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 | 339 | 2021 |
| 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 | 327 | 2011 |
| A comprehensive review of retinal gene therapy SE Boye, SL Boye, AS Lewin, WW Hauswirth Molecular therapy 21 (3), 509-519, 2013 | 323 | 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 | 190 | 2013 |
| 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 | 178 | 2011 |
| 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 | 170 | 2012 |
| 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 20 (8), 824-833, 2013 | 151 | 2013 |
| 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 | 132 | 2012 |
| 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 | 121 | 2014 |
| Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse SE Boye, SL Boye, J Pang, R Ryals, D Everhart, Y Umino, AW Neeley, ... PloS one 5 (6), e11306, 2010 | 112 | 2010 |
| Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa TJ Conlon, WT Deng, K Erger, T Cossette, J Pang, R Ryals, N Clément, ... Human gene therapy Clinical development 24 (1), 23-28, 2013 | 98 | 2013 |
| rAAV2/5 gene-targeting to rods: dose-dependent efficiency and complications associated with different promoters WA Beltran, SL Boye, SE Boye, VA Chiodo, AS Lewin, WW Hauswirth, ... Gene therapy 17 (9), 1162-1174, 2010 | 91 | 2010 |
| Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1 SE Haire, J Pang, SL Boye, I Sokal, CM Craft, K Palczewski, ... Investigative ophthalmology & visual science 47 (9), 3745-3753, 2006 | 88 | 2006 |
| Highly efficient delivery of adeno-associated viral vectors to the primate retina SE Boye, JJ Alexander, CD Witherspoon, SL Boye, JJ Peterson, ME Clark, ... Human gene therapy 27 (8), 580-597, 2016 | 78 | 2016 |
| Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency J Pang, SE Boye, B Lei, SL Boye, D Everhart, R Ryals, Y Umino, B Rohrer, ... Gene therapy 17 (7), 815-826, 2010 | 77 | 2010 |
| Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness ML Williams, JE Coleman, SE Haire, TS Aleman, AV Cideciyan, I Sokal, ... PLoS medicine 3 (6), e201, 2006 | 77 | 2006 |
| Impact of heparan sulfate binding on transduction of retina by recombinant adeno-associated virus vectors SL Boye, A Bennett, ML Scalabrino, KT McCullough, K Van Vliet, ... Journal of virology 90 (8), 4215-4231, 2016 | 76 | 2016 |
| Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque KT McCullough, SL Boye, D Fajardo, K Calabro, JJ Peterson, CE Strang, ... Human gene therapy 30 (5), 571-589, 2019 | 75 | 2019 |
| Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse SL Boye, T Conlon, K Erger, R Ryals, A Neeley, T Cossette, J Pang, ... Investigative ophthalmology & visual science 52 (10), 7098-7108, 2011 | 75 | 2011 |
| Optimization of retinal gene therapy for X-linked retinitis pigmentosa due to RPGR mutations WA Beltran, AV Cideciyan, SE Boye, GJ Ye, S Iwabe, VL Dufour, ... Molecular Therapy 25 (8), 1866-1880, 2017 | 74 | 2017 |
