Patent ID: 9574191
Date: 2017-02-21
CPC Classifications: A61K,C12N

Claim:
1. A method for selectively inhibiting expression of a disease protein encoded by an mRNA having expanded CAG repeat region comprising contacting a cell that produces said disease protein with a double-stranded RNA of 15-30 bases that targets said expanded CAG repeat region of a disease protein mRNA, and said double-stranded RNA further contains 1, 2, 3, 4 or 5 base mismatches as compared to the targeted expanded CAG repeat, wherein (i) inhibiting is selective for said disease protein expression over expression of a normal form of said disease protein, an mRNA for said normal form which lacks said expanded CAG repeat region, (ii) said double-stranded RNA contains no more than one base mismatch in a seed sequence, and (iii) said double-stranded RNA contains the same number of mismatches to the disease protein mRNA as it does to the normal form of said disease protein mRNA, further comprising wherein one strand of the double-stranded RNA has one of the following sequences: GCUGCUGAUGCUGCUGCUGTT (SEQ ID NO 1), GCUGCUGCAGCUGCUGCUGTT (SEQ ID NO 3), GCUGCUGCUACUGCUGCUGTT (SEQ ID NO 5), GCUGCUGCUUCUGCUGCUGTT (SEQ ID NO 7), GCUGCUGCUGAUGCUGCUGTT (SEQ ID NO 9), or UGCUGCUGCAGCUGCUGCUTT (SEQ ID NO 25).