OPTIMIZED STRATEGY FOR MODIFICATIONS THAT SKIP EXONS USING CRISPR / CAS9 WITH TRIPLE GUIDE SEQUENCES

摘要

the genomic edition mediated by crispr / cas9 has clinical potential for the treatment of genetic diseases, such as duchenne muscular dystrophy (dmd), which is caused by mutations in the dystrophin gene. here, the use of three promoters to control the expression of the same dmd guide, a more robust and safer form of genomic editing, was achieved in a humanized mouse model for dmd with a deletion in exon 50 and in a dog ¿ex50 -md.