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.
Claims12
A method of reducing expression of a mutant huntingtin (mHTT) protein in a cell, comprising:
- a. contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.
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 one or more differentiating polymorphisms in the nucleic acid sample;
- c. selecting a nucleic acid silencing agent comprising a sequence that preferentially targets the one or more differentiating polymorphism in the RNA encoding an mHTT protein.
A method of reducing expression of an mHTT protein in a subject, comprising:
- a. obtaining a nucleic acid sample from the subject;
- b. identifying one or more than one differentiating polymorphisms in the nucleic acid sample;
- c. selecting one or more than one nucleic acid silencing agents comprising a sequence that preferentially targets the one or more than one differentiating polymorphism in the RNA encoding an mHTT protein;
- and
- d. administering to the subject an effective amount of the one or more than one nucleic acid silencing agent.
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);
- 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.
Dependencies are inferred from the claim text.