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Human Gene Therapy|July 7, 2026
Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing <i>BEST1</i> Delivered by Subretinal Injection in a Canine Model of Human BestrophinopathyAlexa Gray, Jennifer C Kwok, Yu Sato, et al.
Vision Research|November 30, 2022
Photoreceptor function and structure in retinal degenerations caused by biallelic BEST1 mutationsArtur V Cideciyan, Samuel G Jacobson, Alexander Sumaroka, et al.
Proceedings of the National Academy of Sciences of the United States of America|January 24, 2013
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvementArtur V Cideciyan, Samuel G Jacobson, William A Beltran, et al.
Investigative Ophthalmology & Visual Science|May 17, 2017
Outcome Measures for Clinical Trials of Leber Congenital Amaurosis Caused by the Intronic Mutation in the CEP290 GeneSamuel G Jacobson, Artur V Cideciyan, Alexander Sumaroka, et al.
Human Molecular Genetics|December 17, 2008
ABCA4 disease progression and a proposed strategy for gene therapyArtur V Cideciyan, Malgorzata Swider, Tomas S Aleman, et al.
Human Molecular Genetics|November 1, 2016
Variegated yet non-random rod and cone photoreceptor disease patterns in RPGR-ORF15-associated retinal degenerationJason Charng, Artur V Cideciyan, Samuel G Jacobson, et al.
Plos One|March 7, 2014
Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerationsWilliam A Beltran, Artur V Cideciyan, Karina E Guziewicz, et al.
Plos One|April 25, 2015
Blue cone monochromacy: visual function and efficacy outcome measures for clinical trialsXunda Luo, Artur V Cideciyan, Alessandro Iannaccone, et al.
Investigative Ophthalmology & Visual Science|December 22, 2023
Treatment Strategy With Gene Editing for Late-Onset Retinal Degeneration Caused by a Founder Variant in C1QTNF5Randa T H Li, Alejandro J Roman, Alexander Sumaroka, et al.
Proceedings of the National Academy of Sciences of the United States of America|August 22, 2018
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vectorArtur V Cideciyan, Raghavi Sudharsan, Valérie L Dufour, et al.
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