CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis
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Title
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis
Authors
Keywords
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Journal
Science Advances
Volume 5, Issue 10, Pages eaax1210
Publisher
American Association for the Advancement of Science (AAAS)
Online
2019-10-31
DOI
10.1126/sciadv.aax1210
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