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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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Detalles Bibliográficos
Autores principales:	Cereso, Nicolas, Pequignot, Marie O, Robert, Lorenne, Becker, Fabienne, De Luca, Valerie, Nabholz, Nicolas, Rigau, Valerie, De Vos, John, Hamel, Christian P, Kalatzis, Vasiliki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2014
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362346/ https://www.ncbi.nlm.nih.gov/pubmed/26015956 http://dx.doi.org/10.1038/mtm.2014.11

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