Invention Grant
US08008271B2 Methods and compositions for treating gain-of-function disorders using RNA interference
有权
使用RNA干扰治疗功能障碍功能障碍的方法和组合物
- Patent Title: Methods and compositions for treating gain-of-function disorders using RNA interference
- Patent Title (中): 使用RNA干扰治疗功能障碍功能障碍的方法和组合物
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Application No.: US12175369Application Date: 2008-07-17
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Publication No.: US08008271B2Publication Date: 2011-08-30
- Inventor: Zuoshang Xu , Xugang Xia
- Applicant: Zuoshang Xu , Xugang Xia
- Applicant Address: US MA Boston
- Assignee: University of Massachusetts
- Current Assignee: University of Massachusetts
- Current Assignee Address: US MA Boston
- Agency: Nelson Mullins Riley & Scarborough LLP
- Agent Debra J. Milasincic, Esq.
- Main IPC: A61K31/70
- IPC: A61K31/70 ; C07H21/04
Abstract:
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 agents. The invention also provides for vectors that express RNAi agent and RNAi resistant replacement gene of the present invention.
Public/Granted literature
- US20090042828A1 METHODS AND COMPOSITIONS FOR TREATING GAIN-OF-FUNCTION DISORDERS USING RNA INTERFERENCE Public/Granted day:2009-02-12
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