Article
Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci.
Proceedings of the National Academy of Sciences of the United States of America - 26 Dec 2017
Lessard Samuel, Francioli Laurent, Alfoldi Jessica, Tardif Jean-Claude, Ellinor Patrick T, MacArthur Daniel G, Lettre Guillaume, Orkin Stuart H, Canver Matthew C
Abstract excerpt
The CRISPR-Cas9 nuclease system holds enormous potential for therapeutic genome editing of a wide spectrum of diseases. Large efforts have been made to further understanding of on- and off-target activity to assist the design of CRISPR-based therapies with optimized efficacy and safety. However, current efforts have largely focused on the reference genome or the genome of cell lines to evaluate guide RNA (gRNA)...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.