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Human Gene Therapy|January 8, 2020
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized <i>RPGR</i> Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis PigmentosaValérie L Dufour, Artur V Cideciyan, Guo-Jie Ye, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|June 2, 2017
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR MutationsWilliam A Beltran, Artur V Cideciyan, Shannon E Boye, et al.
Archives of Ophthalmology (Chicago, Ill. : 1960)|September 14, 2011
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsSamuel G Jacobson, Artur V Cideciyan, Ramakrishna Ratnakaram, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 2, 2025
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trialTomas S Aleman, Katherine E Uyhazi, Alejandro J Roman, et al.
Investigative Ophthalmology & Visual Science|December 18, 2024
Retinal Degeneration Associated With Biallelic RDH12 Variants: Longitudinal Evaluation of Retinal Structure and Visual Function in Pediatric PatientsTomas S Aleman, Alejandro J Roman, Katherine E Uyhazi, et al.
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