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US 8,679,750 B2Granted
Methods and compositions for the treatment of Huntington'S disease
AssigneeThe University of British Columbia
InventorsMichael Hayden, Jeffrey Carroll, Simon Warby
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Abstract
Methods and compositions for reducing expression of a mutant huntingtin (mHTT) protein in a cell are provided. Such methods include contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.
Claims8
A method of selecting a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding an mHTT protein of a subject, comprising:
- a. obtaining a nucleic acid sample from the subject;
- b. identifying a differentiating polymorphism in the nucleic acid sample, the differentiating polymorphism is a single nucleotide polymorphism (SNP) identified by RefSNP number rs7685686;
- and
- c. selecting a nucleic acid silencing agent comprising a sequence that preferentially targets the differentiating polymorphism in the RNA encoding an mHTT protein.
1 more element
A method of screening for a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding an mHTT protein in a subject, comprising:
- a. providing a cell heterozygous for a differentiating polymorphism in a nucleic acid sequence encoding huntingtin (HTT), the differentiating polymorphism is a single nucleotide polymorphism (SNP) identified by RefSNP number rs7685686;
- b. contacting the cell with one or more candidate nucleic acid silencing agents targeting the differentiating polymorphism;
- c. assaying the cell for HTT and mHTT RNA, protein or RNA and protein expression;
- and
- d. determining the one or more nucleic acid silencing agents from the candidate nucleic acid silencing agents.
2 more elements
Dependencies are inferred from the claim text.