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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Shranjeno v:
Bibliografske podrobnosti
izdano v:Mol Ther Methods Clin Dev
Main Authors: Brydon, Elizabeth M., Bronstein, Revital, Buskin, Adriana, Lako, Majlinda, Pierce, Eric A., Fernandez-Godino, Rosario
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2019
Teme:
Article
Online dostop: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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