Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa-Reference-Cited by-同舟云学术

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa

Published:2017-08 Issue:8 Volume:25 Page:1854-1865
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Fischer M. Dominik,McClements Michelle E.,Martinez-Fernandez de la Camara Cristina,Bellingrath Julia-Sophia,Dauletbekov Daniyar,Ramsden Simon C.,Hickey Doron G.,Barnard Alun R.,MacLaren Robert E.

Funder

UK Medical Research Council

The Royal College of Surgeons of Edinburgh

Woolf Fisher Trust

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference37 articles.

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3. RPGR: role in the photoreceptor cilium, human retinal disease, and gene therapy;Hosch;Ophthalmic Genet.,2011

4. Phenotypic conservation in patients with X-linked retinitis pigmentosa caused by RPGR mutations;Zahid;JAMA Ophthalmol.,2013

5. Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa;Pawlyk;Gene Ther.,2016

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