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

Internet

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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