Rna-interference by single-stranded rna molecules
First Claim
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1. Use of single-stranded RNA molecule having a length from 14-50 nucleotides wherein at least the 14-20 5′
- most nucleotides are substantially complementary to a target transcript for the manufacture an agent for inhibiting the expression of said target transcript.
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Abstract
The present invention relates to sequence and structural features of single-stranded (ss) RNA molecules required to mediate target-specific nucleic acid modifications by RNA-interference (RNAi), such as target mRNA degradation and/or DNA methylation.
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Citations
31 Claims
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1. Use of single-stranded RNA molecule having a length from 14-50 nucleotides wherein at least the 14-20 5′
- most nucleotides are substantially complementary to a target transcript for the manufacture an agent for inhibiting the expression of said target transcript.
- 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. A pharmaceutical composition for inhibiting the expression of a target transcript by RNAi comprising an active agent a single-stranded RNA molecule having a length from 14-50 nucleotides, wherein at least the 14-20 5′
- most nucleotides are substantially complementary to said target transcript.
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21. A method for the prevention or treatment of disease associated with overexpression of at least one target gene comprising administering a subject in need thereof a single-stranded RNA molecule having a length from 14-50 nucleotides, wherein at least the 14-20 5′
- most nucleotides are substantially complementary to a transcript of said target gene in an amount which is therapeutically effective RNAi.
- 22. Purified human RISC having a molecular weight of from up to about 150-160 kDa.
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26. A host cell or non-human host organism capable of overexpressing RISC.
- 27. A method of enhancing RNAi in a cell or an organism comprising causing said cell or organism to overexpress at least one component of RISC.
- 30. An antisense siRNA precursor molecule in the form of a hairpin stem-loop structure comprising 19 to 29 base pairs in stem, wherein at least 14 nucleotides in the stem are substantially complementary to a target transcript.
Specification