Selective reduction of allelic variants
AssigneeIsis Pharmaceuticals, Inc.
InventorsC. Frank Bennett, Michael Hayden, Susan M. Freier, Sarah Greenlee, Jeffrey Carroll, Simon Warby, Eric E. Swayze
Life of this patent
Estimating the term…
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).
Claims21
-97. (canceled)
A method for determining whether an agent selectively inhibits expression of a first allelic variant of a gene relative to a second allelic variant of said gene wherein said first allelic variant comprises a first nucleotide at a SNP position and said second allelic variant comprises a second nucleotide at said SNP position, comprising:
- contacting a first cell, tissue or animal with said agent at one or more concentrations, wherein said first cell, tissue or animal is homozygous for said first nucleotide at said SNP position;
- contacting a second cell, tissue or animal with said agent at one or more concentrations, wherein said second cell, tissue or animal is homozygous for said second nucleotide at said SNP position or is heterozygous for said first and said second nucleotides at said SNP position;
- measuring inhibition of expression of said allelic variant in each of said cell, tissue or animal for each of said one or more concentrations of said agent;
- and
- comparing said inhibition of expression in each of said cell, tissue or animal for each of said one or more concentrations of said agent to determine whether said agent selectively inhibits expression of said first allelic variant of said gene relative to said second allelic variant of said gene.
Dependencies are inferred from the claim text.