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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Publicado en:Mol Ther Methods Clin Dev
Main Authors: 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: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2014
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4362346/
https://ncbi.nlm.nih.gov/pubmed/26015956
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2014.11
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