公开(公告)号：US20200260698A1

公开(公告)日：2020-08-20

申请号：US16639828

申请日：2018-08-17

Applicant: The Board of Regents of the University of Texas System

Inventor： Viktoriia KYRYCHENKO ,  Eric N. OLSON ,  Rhonda BASSEL-DUBY

IPC: A01K67/027 ,  C12N15/10 ,  C12N15/113 ,  A61P21/00

Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene and absence or deficiency of dystrophin protein in striated muscle. Provided herein are compositions and methods for treating DMD caused by mutations in the dystrophin Actin Binding Domain 1 (ABD-1). The compositions and method described herein can be used to remove mutant sequences in dystrophin ABD-1 to generate a corrected DMD protein that, while lacking one or more exons (e.g., exons 3-9), retains important functional properties.