RNA interference suppression of neurodegenerative diseases and methods of use thereof
First Claim
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1. An isolated RNA duplex comprising a first strand of RNA and a second strand of RNA, wherein the first strand comprises at least 15 contiguous nucleotides encoded by SEQ ID NO:
- 65, and wherein the second strand is complementary to at least 12 contiguous nucleotides of the first strand.
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Abstract
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.
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24 Claims
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1. An isolated RNA duplex comprising a first strand of RNA and a second strand of RNA, wherein the first strand comprises at least 15 contiguous nucleotides encoded by SEQ ID NO:
- 65, and wherein the second strand is complementary to at least 12 contiguous nucleotides of the first strand.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19)
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20. An AAV-1 expressed siRNA comprising an isolated first strand of RNA of 15 to 30 nucleotides in length encoded by SEQ ID NO:
- 65 and an isolated second strand of RNA of 15 to 30 nucleotides in length, wherein the first or second strand comprises a sequence that is complementary to a nucleotide sequence encoding a mutant Huntington'"'"'s Disease protein, wherein at least 12 nucleotides of the first and second strands are complementary to each other and form a small interfering RNA (siRNA) duplex under physiological conditions, and wherein the siRNA silences the expression of the nucleotide sequence encoding the mutant Huntington'"'"'s Disease protein in the cell.
- View Dependent Claims (21, 22, 23, 24)
Specification