RNA interference suppresion of neurodegenerative diseases and methods of use thereof
AssigneeUniversity of Iowa Research Foundation
InventorsBeverly L. Davidson, Haibin Xia, Qinwen Mao, Henry Paulson, Ryan L. Boudreau, Scott Harper
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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.
Claims25
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), or
- (e) shHDEx3.1 21 nt (SEQ ID NO:11),
- and wherein the second strand is complementary to at least 12 contiguous nucleotides of the first strand.
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, wherein the siRNA comprises 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), or
- (e) shHDEx3.1 21 nt (SEQ ID NO:11),
- and wherein the second strand is complementary to at least 12 contiguous nucleotides of the first strand.
Dependencies are inferred from the claim text.