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.
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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