Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient-Reference-Cited by-同舟云学术

Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient

Published:2014 Issue: Volume:1 Page:14011
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Cereso Nicolas,Pequignot Marie O,Robert Lorenne,Becker Fabienne,De Luca Valerie,Nabholz Nicolas,Rigau Valerie,De Vos John,Hamel Christian P,Kalatzis Vasiliki

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference50 articles.

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4. Effect of gene therapy on visual function in Leber's congenital amaurosis;Bainbridge;N Engl J Med,2008

5. Safety and efficacy of gene transfer for Leber's congenital amaurosis;Maguire;N Engl J Med,2008

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