Patent ID: 9458459
Filing Date: 2016-10-04
CPC Classification: A61P,C07K,C12N

Claim Text:
1. A method of treating Duchenne Muscular Dystrophy, comprising the step of administering a therapeutically effective amount of an antisense oligonucleotide that specifically hybridizes to a microRNA binding sequence in a 3′UTR of a utrophin mRNA and inhibits the binding of the microRNA molecule to the 3′ UTR utrophin mRNA, wherein said microRNA is selected from the group consisting of Let-7c, miR-133b, miR-150, miR-196b, miR-206, and miR-296-5p; and wherein the microRNA binding sequence is selected from the group consisting of AGCCATGATCACCTTTCTACCTCA (SEQ ID NO: 18), GTGGGTTGGGGGGCCAA (SEQ ID NO: 17), ATGGGTGGGTGGGTTGGGGG (SEQ ID NO: 16), CCATACCCAGGAAGCACCT (SEQ ID NO: 19), CCACTTTACATTATTACATTCC (SEQ ID NO: 15), ATGGGAAAGTGGGTGGGGGCTTT (SEQ ID NO: 14), and GGGTGGGTGGGTTGGGGGGCC (SEQ ID NO: 23).