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Investigation and Restoration of BEST1 Activity in Patient-derived RPEs with Dominant Mutations

BEST1 is a Ca(2+)-activated Cl(−) channel predominantly expressed in retinal pigment epithelium (RPE), and over 250 genetic mutations in the BEST1 gene have been identified to cause retinal degenerative disorders generally known as bestrophinopathies. As most BEST1 mutations are autosomal dominant,...

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Salvato in:
Dettagli Bibliografici
Pubblicato in:Sci Rep
Autori principali: Ji, Changyi, Li, Yao, Kittredge, Alec, Hopiavuori, Austin, Ward, Nancy, Yao, Peng, Fukuda, Yohta, Zhang, Yu, Tsang, Stephen H., Yang, Tingting
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6910965/
https://ncbi.nlm.nih.gov/pubmed/31836750
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-54892-7
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