Optimized Strategy for Exon Skipping Modifications with CRISPR / CAS9 with Triple Guide Sequences

摘要

CRISPR / Cas9-mediated genome editing retains clinical efficacy for treating genetic diseases such as Ducken's muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. Here we use three promoters to drive the expression of the same DMD guide RNA, which results in a more robust and safe form of genome editing in a humanized mouse model for DMD with an exon 50 deletion, and in ΔEx50-MD dogs. Achieved.