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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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| Veröffentlicht in: | Mol Ther |
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| Hauptverfasser: | , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Nature Publishing Group
2015
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| Schlagworte: | |
| Online Zugang: | 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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