Ionis Pharmaceuticals, Inc.
Selective Reduction of Allelic Variants
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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).
Status:
Application
Type:
Utility
Filling date:
14 Jan 2020
Issue date:
3 Dec 2020