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Lancet (London, England)|September 7, 2024
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation studyPaul Yang, Laura P Pardon, Allen C Ho, et al.Proceedings of the National Academy of Sciences of the United States of America|September 24, 2008
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kineticsArtur V Cideciyan, Tomas S Aleman, Sanford L Boye, et al.Human Gene Therapy|September 1, 2006
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosisSamuel G Jacobson, Sanford L Boye, Tomas S Aleman, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|April 29, 2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injectionSamuel G Jacobson, Gregory M Acland, Gustavo D Aguirre, et al.Human Gene Therapy|December 7, 2011
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of conceptAstra Dinculescu, Jackie Estreicher, Juan C Zenteno, 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.Nature Medicine|December 19, 2018
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defectArtur V Cideciyan, Samuel G Jacobson, Arlene V Drack, et al.Nature Medicine|April 5, 2022
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trialStephen R Russell, Arlene V Drack, Artur V Cideciyan, et al.Pageof 11