Safety and efficacy of gene transfer for Leber's congenital amaurosis AM Maguire, F Simonelli, EA Pierce, EN Pugh Jr, F Mingozzi, J Bennicelli, ... New England Journal of Medicine 358 (21), 2240-2248, 2008 | 2587 | 2008 |
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle S Yu, E Lee, D Bok, D Hamasaki, N Chen, P Goletz, JX Ma, RK Crouch, ... Nature genetics 20 (4), 344-351, 1998 | 1102 | 1998 |
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial AM Maguire, KA High, A Auricchio, JF Wright, EA Pierce, F Testa, ... The Lancet 374 (9701), 1597-1605, 2009 | 1043 | 2009 |
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration F Simonelli, AM Maguire, F Testa, EA Pierce, F Mingozzi, JL Bennicelli, ... Molecular Therapy 18 (3), 643-650, 2010 | 723 | 2010 |
Mutations in RPE65 cause Leber's congenital amaurosis F Marlhens, C Bareil, JM Griffoin, E Zrenner, P Amalric, C Eliaou, SY Liu, ... Nature genetics 17 (2), 139-141, 1997 | 708 | 1997 |
Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle TM Redmond, E Poliakov, S Yu, JY Tsai, Z Lu, S Gentleman Proceedings of the National Academy of Sciences 102 (38), 13658-13663, 2005 | 463 | 2005 |
Molecular cloning and expression of RPE65, a novel retinal pigment epithelium-specific microsomal protein that is post-transcriptionally regulated in vitro CP Hamel, E Tsilou, BA Pfeffer, JJ Hooks, B Detrick, TM Redmond Journal of Biological Chemistry 268 (21), 15751-15757, 1993 | 401 | 1993 |
Identification, expression, and substrate specificity of a mammalian β-carotene 15, 15′-dioxygenase TM Redmond, S Gentleman, T Duncan, S Yu, B Wiggert, E Gantt, ... Journal of Biological Chemistry 276 (9), 6560-6565, 2001 | 364 | 2001 |
Protection of Rpe65-deficient mice identifies rhodopsin as a mediator of light-induced retinal degeneration C Grimm, A Wenzel, F Hafezi, S Yu, TM Redmond, CE Remé Nature genetics 25 (1), 63-66, 2000 | 350 | 2000 |
Intrachoroidal neovascularization in transgenic mice overexpressing vascular endothelial growth factor in the retinal pigment epithelium C Schwesinger, C Yee, RM Rohan, AM Joussen, A Fernandez, TN Meyer, ... The American journal of pathology 158 (3), 1161-1172, 2001 | 315 | 2001 |
Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog K Narfström, ML Katz, R Bragadottir, M Seeliger, A Boulanger, ... Investigative ophthalmology & visual science 44 (4), 1663-1672, 2003 | 290 | 2003 |
New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis MW Seeliger, C Grimm, F Ståhlberg, C Friedburg, G Jaissle, E Zrenner, ... Nature genetics 29 (1), 70-74, 2001 | 254 | 2001 |
In utero gene therapy rescues vision in a murine model of congenital blindness NS Dejneka, EM Surace, TS Aleman, AV Cideciyan, A Lyubarsky, ... Molecular Therapy 9 (2), 182-188, 2004 | 232 | 2004 |
Uveoretinitis and pinealitis induced by immunization with interphotoreceptor retinoid-binding protein. I Gery, B Wiggert, TM Redmond, T Kuwabara, MA Crawford, BP Vistica, ... Investigative ophthalmology & visual science 27 (8), 1296-1300, 1986 | 224 | 1986 |
Spontaneous activity of opsin apoprotein is a cause of Leber congenital amaurosis ML Woodruff, Z Wang, HY Chung, TM Redmond, GL Fain, J Lem Nature genetics 35 (2), 158-164, 2003 | 212 | 2003 |
Spectral domain optical coherence tomography in mouse models of retinal degeneration G Huber, SC Beck, C Grimm, A Sahaboglu-Tekgoz, F Paquet-Durand, ... Investigative ophthalmology & visual science 50 (12), 5888-5895, 2009 | 209 | 2009 |
Isolation and characterization of monkey interphotoreceptor retinoid-binding protein, a unique extracellular matrix component of the retina TM Redmond, B Wiggert, FA Robey, NN Yen, MS Lewis, L Lee, ... Biochemistry 24 (3), 787-793, 1985 | 178 | 1985 |
Effect of Rpe65 knockout on accumulation of lipofuscin fluorophores in the retinal pigment epithelium ML Katz, TM Redmond Investigative ophthalmology & visual science 42 (12), 3023-3030, 2001 | 174 | 2001 |
Appropriately differentiated ARPE-19 cells regain phenotype and gene expression profiles similar to those of native RPE cells W Samuel, C Jaworski, OA Postnikova, RK Kutty, T Duncan, LX Tan, ... Molecular vision 23, 60, 2017 | 170 | 2017 |
Interphotoreceptor retinoid-binding protein: gene characterization, protein repeat structure, and its evolution DE Borst, TM Redmond, JE Elser, MA Gonda, B Wiggert, GJ Chader, ... Journal of Biological Chemistry 264 (2), 1115-1123, 1989 | 154 | 1989 |