Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes-Reference-Cited by-同舟云学术

Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes

Published:2023-12 Issue: Volume:32 Page:101873
ISSN:2451-9936
Container-title:American Journal of Ophthalmology Case Reports
language:en
Short-container-title:American Journal of Ophthalmology Case Reports

Author:

Cideciyan Artur V.^ORCID,Jacobson Samuel G.,Ho Allen C.,Swider Malgorzata,Sumaroka Alexander,Roman Alejandro J.^ORCID,Wu Vivian,Russell Robert C.,Viarbitskaya Iryna,Garafalo Alexandra V.,Schwartz Michael R.,Girach Aniz

Funder

Research to Prevent Blindness

ProQR Therapeutics

Publisher

Elsevier BV

Subject

Ophthalmology

Reference56 articles.

1. The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders;Bramall;Annu Rev Neurosci,2010

2. Non-syndromic retinitis pigmentosa;Verbakel;Prog Retin Eye Res,2018

3. Progress in treating inherited retinal diseases: early subretinal gene therapy clinical trials and candidates for future initiatives;Garafalo;Prog Retin Eye Res,2020

4. Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions;Kumaran;Br J Ophthalmol,2017

5. Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosis;den Hollander;Am J Hum Genet,2006

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