Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci

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, cur...

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