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

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

Inherited retinal dystrophies (IRDs) comprise a large group of genetically and clinically heterogeneous diseases that lead to progressive vision loss, for which a paucity of disease-mimicking animal models renders preclinical studies difficult. We sought to develop pertinent human cellular IRD model...

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Bibliographic Details
Main Authors: Nicolas Cereso, Marie O Pequignot, Lorenne Robert, Fabienne Becker, Valerie De Luca, Nicolas Nabholz, Valerie Rigau, John De Vos, Christian P Hamel, Vasiliki Kalatzis
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116300778

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http://www.sciencedirect.com/science/article/pii/S2329050116300778

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