- 专利标题： Genetic and pharmacological transcriptional upregulation of the repressed FXN gene as a therapeutic strategy for Friedreich ataxia
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申请号： US17406441申请日： 2021-08-19
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公开(公告)号： US11873494B2公开(公告)日： 2024-01-16
- 发明人: Michael R. Green , Minggang Fang
- 申请人： University of Massachusetts
- 申请人地址： US MA Boston
- 专利权人： University of Massachusetts
- 当前专利权人： University of Massachusetts
- 当前专利权人地址： US MA Boston
- 代理机构： Fish & Richardson P.C.
- 分案原申请号： US16487876
- 主分类号： C07K14/47
- IPC分类号： C07K14/47 ; C12N15/113 ; A61K45/06
摘要：
Methods of treating subjects having disorders associated with mutations in the FXN gene and/or having reduced expression of frataxin protein, e.g., Friedreich ataxia (FA). Generally, the methods include administering a therapeutically effective amount of an agent that increases expression of frataxin protein as described herein, e.g., an inhibitor of a FXN Repressing Factor (FXN-RF).
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