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RNA interference for the treatment of gain-of-function disorders

  • US 9,434,943 B2
  • Filed: 01/22/2014
  • Issued: 09/06/2016
  • Est. Priority Date: 09/12/2003
  • Status: Active Grant
First Claim
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1. A composition for treating a subject having or at risk for Huntington'"'"'s disease caused by a mutation within the htt gene encoding a gain-of-function mutant huntingtin protein, comprising:

  • a vector that expresses an effective amount of an siRNA or an shRNA targeting a non-disease causing heterozygous single nucleotide polymorphism (SNP) within a gene encoding said mutant huntingtin protein located at a site distinct from the expanded CAG region mutation within the htt gene encoding the mutant huntingtin protein.

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