Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial-Reference-Cited by-同舟云学术

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Published:2016-08 Issue:10045 Volume:388 Page:661-672
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Bennett Jean,Wellman Jennifer,Marshall Kathleen A,McCague Sarah,Ashtari Manzar,DiStefano-Pappas Julie,Elci Okan U,Chung Daniel C,Sun Junwei,Wright J Fraser,Cross Dominique R,Aravand Puya,Cyckowski Laura L,Bennicelli Jeannette L,Mingozzi Federico,Auricchio Alberto,Pierce Eric A,Ruggiero Jason,Leroy Bart P,Simonelli Francesca,High Katherine A,Maguire Albert M

Funder

FWO Flanders

Publisher

Elsevier BV

Subject

General Medicine

Reference30 articles.

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2. Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration;Thompson;Invest Ophthalmol Vis Sci,2000

3. Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle;Redmond;Nat Genet,1998

4. Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle;Redmond;Proc Natl Acad Sci USA,2005

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

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