Patent ID: 9006198
Filing Date: 2015-04-14
Classification: A61P,C12N,C12Q

Abstract:
1. A method of selectively reducing the expression of a mutant huntingtin allele in a cell, tissue, or animal comprising administering to the cell, tissue, or animal a compound comprising a modified, single-stranded antisense oligonucleotide complementary to the mutant huntingtin allele at a position comprising a single nucleotide polymorphism, wherein the modified oligonucleotide consists of 12 to 30 linked nucleosides and 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, wherein the expression of the mutant huntingtin allele is selectively reduced, and wherein the single nucleotide polymorphism is rs362306, rs362331, rs2298969, rs7685686, rs4690072, rs2024115, or rs363088.