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    Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

    Leber congenital amaurosis type 10 is a severe retinal dystrophy caused by mutations in the CEP290 gene1,2. We developed EDIT-101, a candidate genome-editing therapeutic, to remove the aberrant splice donor creat...

    Morgan L. Maeder, Michael Stefanidakis, Christopher J. Wilson… in Nature Medicine (2019)