| Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector AV Cideciyan, R Sudharsan, VL Dufour, MT Massengill, S Iwabe, ... Proceedings of the National Academy of Sciences 115 (36), E8547-E8556, 2018 | 131 | 2018 |
| 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 | 81 | 2017 |
| BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure SGJGDA Karina E. Guziewicz, Artur V. Cideciyan, William A. Beltran, András M ... PNAS, 2018 | 77* | 2018 |
| Long-term structural outcomes of late-stage RPE65 gene therapy KL Gardiner, AV Cideciyan, M Swider, VL Dufour, A Sumaroka, ... Molecular Therapy 28 (1), 266-278, 2020 | 62 | 2020 |
| scAAVengr, a transcriptome-based pipeline for quantitative ranking of engineered AAVs with single-cell resolution BE Öztürk, ME Johnson, M Kleyman, S Turunç, J He, S Jabalameli, Z Xi, ... Elife 10, e64175, 2021 | 38 | 2021 |
| Overlap of abnormal photoreceptor development and progressive degeneration in Leber congenital amaurosis caused by NPHP5 mutation LM Downs, EM Scott, AV Cideciyan, S Iwabe, V Dufour, KL Gardiner, ... Human molecular genetics 25 (19), 4211-4226, 2016 | 34 | 2016 |
| Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X … VL Dufour, AV Cideciyan, G Ye, C Song, A Timmers, PL Habecker, W Pan, ... Human Gene Therapy 31 (3-4), 253-267, 2020 | 25 | 2020 |
| Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis GD Aguirre, AV Cideciyan, VL Dufour, A Ripolles-García, R Sudharsan, ... Molecular Therapy 29 (8), 2456-2468, 2021 | 19 | 2021 |
| Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy C Song, VL Dufour, AV Cideciyan, GJ Ye, M Swider, JA Newmark, ... Human Gene Therapy 31 (13-14), 743-755, 2020 | 19 | 2020 |
| Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness K Miyadera, E Santana, K Roszak, S Iffrig, M Visel, S Iwabe, RF Boyd, ... Proceedings of the National Academy of Sciences 119 (13), e2117038119, 2022 | 15 | 2022 |
| Focal/multifocal and geographic retinal dysplasia in the dog—In vivo retinal microanatomy analyses S Iwabe, VL Dufour, JM Guzmán, DM Holle, JA Cohen, WA Beltran, ... Veterinary ophthalmology 23 (2), 292-304, 2020 | 11 | 2020 |
| Effect of topical ophthalmic dorzolamide (2%)–timolol (0.5%) solution and ointment on intraocular pressure in normal horses KL Tofflemire, EM Whitley, AM Flinn, VL Dufour, G Ben‐Shlomo, ... Veterinary ophthalmology 18 (6), 457-461, 2015 | 8 | 2015 |
| Underdeveloped RPE apical domain underlies lesion formation in canine bestrophinopathies KE Guziewicz, E McTish, VL Dufour, K Zorych, A Dhingra, ... Retinal Degenerative Diseases: Mechanisms and Experimental Therapy, 309-315, 2018 | 7 | 2018 |
| In-vivo longitudinal changes in thickness of the postnatal canine retina VL Dufour, Y Yu, W Pan, GS Ying, GD Aguirre, WA Beltran Experimental eye research 192, 107926, 2020 | 6 | 2020 |
| scAAVengr: Single-cell transcriptome-based quantification of engineered AAVs in non-human primate retina BE Öztürk, ME Johnson, M Kleyman, S Turunç, J He, S Jabalameli, Z Xi, ... BioRxiv, 2020.10. 01.323196, 2020 | 3 | 2020 |
| Long-term longitudinal assessment of functional rescue in mutant XLRP dogs after RPGR gene augmentation therapy WA Beltran, VL Dufour, SM Ainsworth, K Roszak, GD Aguirre Investigative Ophthalmology & Visual Science 60 (9), 4031-4031, 2019 | 2 | 2019 |
| Isolated population helps tease out a third locus underlying a multigenic form of canine RPGRIP1 cone-rod dystrophy K Miyadera, L Murgiano, C Spector, FP Marinho, V Dufour, RG Das, ... Investigative Ophthalmology & Visual Science 59 (9), 1438-1438, 2018 | 2 | 2018 |
| Evaluation of AAV2tYF-GRK1-RPGR vectors in a canine model of RPGR-XLRP G Ye, WA Beltran, VL Dufour, FP Marinho, I Martynyuk, C Song, D Knop, ... Investigative Ophthalmology & Visual Science 58 (8), 4486-4486, 2017 | 2 | 2017 |
| Long-term preservation of photoreceptor function and structure following early-stage treatment by AAV-mediated gene augmentation in canine model of NPHP5 Leber congenital amaurosis GD Aguirre, AV Cideciyan, SL Boye, S Iwabe, V Dufour, M Swider, ... Investigative Ophthalmology & Visual Science 59 (9), 6006-6006, 2018 | 1 | 2018 |
| Directed evolution of AAV vectors guided by deep sequencing creates variants that bypass structural barriers in canine retina LC Byrne, M Visel, VL Dufour, FP Marinho, GD Aguirre, WA Beltran, ... MOLECULAR THERAPY 25 (5), 232-233, 2017 | 1 | 2017 |
William BeltranProfessor of Ophthalmology- University of PennsylvaniaVerified email at vet.upenn.edu
