Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates-Reference-Cited by-同舟云学术

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Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates

Published:2019-12 Issue: Volume:15 Page:133-148
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Weed Lindsey,Ammar Michael J.,Zhou Shangzhen,Wei Zhangyong,Serrano Leona W.,Sun Junwei,Lee Vivian,Maguire Albert M.,Bennett Jean,Aleman Tomas S.

Funder

Center for Advanced Retinal and Ocular Therapeutics

The Brenda and Matthew Shapiro Stewardship

Robert and Susan Heidenberg Investigative Research Fund for Ocular Gene Therapy

The Pennsylvania Lions Sight Conservation and Research Foundation

The Paul and Evanina Bell Mackall Foundation Trust

Research to Prevent Blindness

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference60 articles.

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

2. Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait;Wright;Nat. Rev. Genet.,2010

3. Leber Congenital Amaurosis;Weleber,2013

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. Genetic analysis of RPE65: from human disease to mouse model;Redmond;Methods Enzymol.,2000

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4. Lessons Learned from the Development of the First FDA-Approved Gene Therapy Drug, Voretigene Neparvovec-rzyl;Cold Spring Harbor Perspectives in Medicine;2022-09-27

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