Selective Reduction of Allelic Variants
AssigneesIonis Pharmaceuticals, Inc., The University of British Columbia
InventorsC. Frank Bennett, Michael Hayden, Susan M. Freier, Sarah Greenlee, Jeffrey Carroll, Simon Warby, Eric E. Swayze
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Abstract
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).
Claims19
A method of treating Huntington's Disease, comprising selectively reducing expression of a mutant huntingtin allele in an animal in need thereof, comprising administering to the animal a compound, comprising a modified oligonucleotide consisting of 15 to 20 linked nucleosides and fully complementary to the mutant huntingtin allele, wherein position 5, 6, 7, 8, 9, 10, 11, or 12 of the modified oligonucleotide, as counted from the 5′ terminus of the modified oligonucleotide, aligns with the SNP rs362272, and wherein the modified oligonucleotide comprises a wing-gap-wing motif.
Dependencies are inferred from the claim text.