发明名称 |
METHODS AND COMPOSITIONS FOR TREATING GAIN-OF-FUNCTION DISORDERS USING RNA INTERFERENCE |
摘要 |
The present invention relates to novel methods for treating dominant gain-of-function diseases. The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis (ALS), with RNAi agent. The invention further provides RNAi resistant replacement genes containing mismatches with their respective RNAi agent's. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention. |
申请公布号 |
WO2005096781(A2) |
申请公布日期 |
2005.10.20 |
申请号 |
WO2005US11983 |
申请日期 |
2005.04.06 |
申请人 |
UNIVERSITY OF MASSACHUSETTS;XU, ZUOSHANG;XIA, XUGANG |
发明人 |
XU, ZUOSHANG;XIA, XUGANG |
分类号 |
A61K48/00;C07H21/02;C12N9/02;C12N15/11;C12N15/113;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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