Title:
NICKED OR GAPPED NUCLEIC ACID MOLECULES AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO2008049078
Kind Code:
B1
Abstract:
The present disclosure provides meroduplex (nicked or gapped) ribonucleic acid molecules (mdRNA) that decreases or silences target gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target gene RNA. In addition, the meroduplex may have one or more modifications or substitutions, such as nucleotide base, sugar, terminal cap structure, internucleotide linkage, or any combination of such modifications. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease related to altered expression of a target gene.
Inventors:
QUAY STEVEN C (US)
MCSWIGGEN JAMES (US)
VAISH NARENDRA K (US)
AHMADIAN MOHAMMAD (US)
MCSWIGGEN JAMES (US)
VAISH NARENDRA K (US)
AHMADIAN MOHAMMAD (US)
Application Number:
PCT/US2007/081836
Publication Date:
June 26, 2008
Filing Date:
October 18, 2007
Export Citation:
Assignee:
NASTECH PHARM CO (US)
QUAY STEVEN C (US)
MCSWIGGEN JAMES (US)
VAISH NARENDRA K (US)
AHMADIAN MOHAMMAD (US)
QUAY STEVEN C (US)
MCSWIGGEN JAMES (US)
VAISH NARENDRA K (US)
AHMADIAN MOHAMMAD (US)
International Classes:
A61K31/7105; C12N15/11
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