Article
CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Feb 2017
Ruan Guo-Xiang, Barry Elizabeth, Yu Dan, Lukason Michael, Cheng Seng H, Scaria Abraham
Abstract excerpt
As the most common subtype of Leber congenital amaurosis (LCA), LCA10 is a severe retinal dystrophy caused by mutations in the CEP290 gene. The most frequent mutation found in patients with LCA10 is a deep intronic mutation in CEP290 that generates a cryptic splice donor site. The large size of the CEP290 gene prevents its use in adeno-associated virus (AAV)-mediated gene augmentation therapy. Here, we show that...
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