RNA INTERFERENCE SUPPRESION OF NEURODEGENERATIVE DISEASES AND METHODS OF USE THEREOF
First Claim
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1. An 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(a) shHDEx2.1 (SEQ ID NO:
- 7),(b) shHDEx2.2 19 nt (SEQ ID NO;
8),(c) shHDEx2.2 21 nt (SEQ ID NO;
9),(d) shHDEx3.1 19 nt (SEQ ID NO;
10),(e) shHDEx3.1 21 nt (SEQ ID NO;
11),(f) siEX58#1 (SEQ ID NO;
12),(g) siEX58#2 (SEQ ID NO;
17),(h) shSCA1.F10 (SEQ ID NO;
13), or(i) shSCA1.F11 (SEQ ID NO;
14),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.
81 Citations
26 Claims
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1. An 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
(a) shHDEx2.1 (SEQ ID NO: - 7),
(b) shHDEx2.2 19 nt (SEQ ID NO;
8),(c) shHDEx2.2 21 nt (SEQ ID NO;
9),(d) shHDEx3.1 19 nt (SEQ ID NO;
10),(e) shHDEx3.1 21 nt (SEQ ID NO;
11),(f) siEX58#1 (SEQ ID NO;
12),(g) siEX58#2 (SEQ ID NO;
17),(h) shSCA1.F10 (SEQ ID NO;
13), or(i) shSCA1.F11 (SEQ ID NO;
14),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)
- 7),
- 17. A viral vector comprising a promoter and an miRNA shuttle containing an embedded siRNA that specifically targets a sequence associated with a condition amenable to siRNA therapy.
Specification