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AAV-Mediated Gene Augmentation Therapy Restores Critical Functions in Mutant PRPF31(+/−) iPSC-Derived RPE Cells

Retinitis pigmentosa (RP) is the most common form of inherited vision loss and is characterized by degeneration of retinal photoreceptor cells and the retinal pigment epithelium (RPE). Mutations in pre-mRNA processing factor 31 (PRPF31) cause dominant RP via haploinsufficiency with incomplete penetr...

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Detalles Bibliográficos
Autores principales:	Brydon, Elizabeth M., Bronstein, Revital, Buskin, Adriana, Lako, Majlinda, Pierce, Eric A., Fernandez-Godino, Rosario
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909184/ https://www.ncbi.nlm.nih.gov/pubmed/31890732 http://dx.doi.org/10.1016/j.omtm.2019.10.014

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