BEST1: the Best Target for Gene and Cell Therapies

A retinal pigmented epithelial (RPE) disorder, bestrophinopathy has recently been proven to be amenable to gene and cell-based therapies in preclinical models. RPE disorders and allied retinal degenerations exhibit significant genetic heterogeneity, and diverse mutations can result in similar diseas...

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Bibliographic Details
Published in:Mol Ther
Main Authors: Yang, Tingting, Justus, Sally, Li, Yao, Tsang, Stephen H
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2015
Subjects:
Review
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4700113/
https://ncbi.nlm.nih.gov/pubmed/26388462
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.177
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