ARTICLE | Translation in Brief
Keeping CRISPR on target
How Editas is using machine learning to predict off-target effects of CRISPR
September 13, 2018 5:36 PM UTC
Editas Medicine Inc. (NASDAQ:EDIT) has developed an in silico model of Staphylococcus aureus Cas9 (SaCas9) gene editing to help it select guide RNA sequences with minimal risk of off-target effects.
Editas’ lead candidate EDIT-101 is built on SaCas9. The preclinical therapy is in development for Leber congenital amaurosis type 10 (LCA10) and uses SaCas9 to remove or inverse the IVS26 mutation in the centrosomal protein 290kDa (CEP290). ...
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