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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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Publicado no: | Mol Ther Methods Clin Dev |
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Main Authors: | , , , , , , , , , |
Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
Nature Publishing Group
2014
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Assuntos: | |
Acesso em linha: | 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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