Patent ID: 9457102
Filing Date: 2016-10-04
CPC Classification: A61K,A61P,C12N

Claim Text:
1. A method for treating Duchenne muscular dystrophy in a subject in need thereof, the method comprising: administering a Bubble liposome and a morpholino oligomer bound to a surface of the Bubble liposome into a blood vessel of the subject, and transcutaneously irradiating a muscle tissue with ultrasound, wherein the morpholino oligomer in a splicing process for a pre-mRNA of a dystrophin gene having a stop codon resulting from a mutation, produces a mature mRNA skipping an exon having the mutation therein; the morpholino oligomer comprises a base sequence complementary to a region comprising a splicing enhancer sequence for said exon having the mutation therein; and the morpholino oligomer has a polymerization degree of from 15 to 50 mer.