Patent ID: 8957040
Filing Date: 2015-02-17
Classification: A61P,C12N

Abstract:
1. A compound comprising a modified antisense oligonucleotide consisting of 12 to 30 linked nucleosides targeted to a single nucleotide polymorphism site, wherein the modified oligonucleotide comprises a wing-gap-wing motif with a 5′ wing region positioned at the 5′ end of a deoxynucleoside gap, and a 3′ wing region positioned at the 3′ end of the deoxynucleoside gap, wherein position 8, 9, or 10 of the modified oligonucleotide, as counted from the 5′ terminus of the modified oligonucleotide, aligns with the single nucleotide polymorphism, and wherein the single nucleotide polymorphism is on a mutant allele that is associated with Huntington's Disease.