发明名称 |
DUAL FUNCTIONAL OLIGONUCLEOTIDES FOR USE IN REPRESSING MUTANT GENE EXPRESSION |
摘要 |
The present invention is based, in part, on the discovery that endogenous miRNAs can be recruited for translational repression of target mRNAs. The RNA-silencing agents and the methods described herein, thereby provide a means by which to treat genetic (e.g., genetic neurodegenerative diseases such as Huntington's Disease) or non-genetic diseases by, for example, blocking the synthesis of proteins that contribute to the diseases. Accordingly the RNA-silencing agents of the present invention have an mRNA targeting moiety, a linking moiety, and an miRNA recruiting moiety. |
申请公布号 |
WO2005078096(A3) |
申请公布日期 |
2005.12.22 |
申请号 |
WO2005US04206 |
申请日期 |
2005.02.09 |
申请人 |
UNIVERSITY OF MASSACHUSETTS;ZAMORE, PHILLIP, D.;ARONIN, NEIL;BRODERICK, JENNIFER |
发明人 |
ZAMORE, PHILLIP, D.;ARONIN, NEIL;BRODERICK, JENNIFER |
分类号 |
A61K48/00;C07H21/02;C12N15/113;C12N15/63;C12N15/85;C12Q1/68 |
主分类号 |
A61K48/00 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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