Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives

Author:

Garafalo Alexandra V.,Cideciyan Artur V.,Héon Elise,Sheplock Rebecca,Pearson Alexander,WeiYang Yu CaberryORCID,Sumaroka Alexander,Aguirre Gustavo D.,Jacobson Samuel G.ORCID

Funder

National Institutes of Health/National Eye Institute

Publisher

Elsevier BV

Subject

Sensory Systems,Ophthalmology

Reference239 articles.

1. Gene therapy restores vision in a canine model of childhood blindness;Acland;Nat. Genet.,2001

2. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness;Acland;Mol. Ther.,2005

3. Retinal degenerations in the dog. I. Rod dysplasia;Aguirre;Exp. Eye Res.,1978

4. Natural history of the central structural abnormalities in choroideremia: a prospective cross-sectional study;Aleman;Ophthalmology,2017

5. AAV2-hCHM subretinal delivery to the macular in choroideremia: 2 year results of an ongoing phase I/II gene therapy trial;Aleman;Investig. Ophthalmol. Vis. Sci.,2019

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