METHODS AND COMPOSITIONS FOR THE TREATMENT OF HUNTINGTON'S DISEASE
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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.
12 Citations
15 Claims
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1. (canceled)
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2. A method of selecting a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding an mHTT protein of a subject, comprising:
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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 identified by RefSNP number rs363102; and c. selecting a nucleic acid silencing agent comprising a sequence that preferentially targets the differentiating polymorphism in the RNA encoding an mHTT protein. - View Dependent Claims (6, 7, 8)
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3. (canceled)
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4. A method of screening for a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding an mHTT protein in a subject, comprising:
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d. 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 rs363102; e. contacting the cell with one or more candidate nucleic acid silencing agents targeting the differentiating polymorphism; f. assaying the cell for HTT and mHTT RNA, protein or RNA and protein expression; and g. determining the one or more nucleic acid silencing agents from the candidate nucleic acid silencing agents. - View Dependent Claims (13, 14, 15)
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5. (canceled)
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9-12. -12. (canceled)
Specification