Patent ID: 11926829
Assignee: INSERM (INSTITUT NATIONAL DE LA SANTé ET DE LA RECHERCHE MéDICALE)
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC C

Claim 0:
1. An allele specific siRNA (AS-siRNA) that comprises a sequence mismatch and is able to silence the expression of only one allele of a heterozygous DNM2 gene in a cell, wherein the DNM2 gene is heterozygous for the presence of a disease-causing mutation, wherein said AS-siRNA targets a region of a human DNM2 gene transcript comprising said disease-causing mutation, wherein the disease-causing mutation is 1393C>T; c.1105C>T, c.1106G>A, c.1856C>T or c.1948G>A, and wherein the AS-siRNA comprises a sense strand and an antisense strand and the antisense strand only contains bases selected from the group consisting of guanine, cytosine, adenine, uracil, and deoxythymidine.