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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Brydon, Elizabeth M., Bronstein, Revital, Buskin, Adriana, Lako, Majlinda, Pierce, Eric A., Fernandez-Godino, Rosario
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6909184/
https://ncbi.nlm.nih.gov/pubmed/31890732
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.10.014
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