Reversible siRNA-Based Silencing of Mutant and Endogenous Wild-Type Huntingtin Gene and its Application for the Treatment of Huntington's Disease
First Claim
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1. An isolated double-stranded short interfering nucleic acid molecule comprising complementary sense and antisense regions, wherein:
- the antisense region has 15 to no more than 19 contiguous nucleotides that are complementary to a human huntingtin transcript, said nucleotides being encoded by a sequence selected from the group consisting of SEQ ID NO;
1, SEQ ID NO;
2, and SEQ ID NO;
3,the sense and antisense regions have at least 15 contiguous nucleotides that are complementary to each other and form a duplex, andsaid double-stranded short interfering nucleic acid molecule inhibits the expression of endogenous wild-type and exogenous human mutant huntingtin genes in cells of a non-human mammal which are expressing both said huntingtin genes.
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Abstract
Isolated double-stranded short interfering nucleic acid molecules inhibiting the expression of endogenous wild-type and exogenous human mutant huntintin genes in cells of a non-human mammal which are expressing both said huntingtin genes, and their application for the treatment of Huntington'"'"'s disease as well as to study Huntington'"'"'s disease in rodent models.
28 Citations
20 Claims
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1. An isolated double-stranded short interfering nucleic acid molecule comprising complementary sense and antisense regions, wherein:
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the antisense region has 15 to no more than 19 contiguous nucleotides that are complementary to a human huntingtin transcript, said nucleotides being encoded by a sequence selected from the group consisting of SEQ ID NO;
1, SEQ ID NO;
2, and SEQ ID NO;
3,the sense and antisense regions have at least 15 contiguous nucleotides that are complementary to each other and form a duplex, and said double-stranded short interfering nucleic acid molecule inhibits the expression of endogenous wild-type and exogenous human mutant huntingtin genes in cells of a non-human mammal which are expressing both said huntingtin genes. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20)
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Specification