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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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Brydon, Elizabeth M., Bronstein, Revital, Buskin, Adriana, Lako, Majlinda, Pierce, Eric A., Fernandez-Godino, Rosario
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Article
Acceso en línea: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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