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Article

BEST1: the Best Target for Gene and Cell Therapies

21 Sept 2015

Abstract excerpt

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 disease phenotypes. Several RPE disorders have recently become targets for gene therapies in humans. The year 2011 brought...

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PMID: 26388462
DOI: 10.1038/mt.2015.177

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