Invention Grant
- Patent Title: Genetic and pharmacological transcriptional upregulation of the repressed FXN gene as a therapeutic strategy for Friedreich ataxia
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Application No.: US16487876Application Date: 2018-02-14
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Publication No.: US11124795B2Publication Date: 2021-09-21
- Inventor: Michael R. Green , Minggang Fang
- Applicant: University of Massachusetts
- Applicant Address: US MA Boston
- Assignee: University of Massachusetts
- Current Assignee: University of Massachusetts
- Current Assignee Address: US MA Boston
- Agency: Fish & Richardson P.C.
- International Application: PCT/US2018/018181 WO 20180214
- International Announcement: WO2018/160356 WO 20180907
- Main IPC: C07K14/47
- IPC: C07K14/47 ; C12N15/113 ; A61K45/06
Abstract:
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).
Information query
