Invention Grant
- Patent Title: Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
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Application No.: US15856653Application Date: 2017-12-28
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Publication No.: US11564977B2Publication Date: 2023-01-31
- Inventor: Martin K. Childers , Alan H. Beggs , Ana Maria Buj Bello
- Applicant: Wake Forest University Health Sciences , Genethon , Children's Medical Center Corporation
- Applicant Address: US NC Winston-Salem; FR Evry; US MA Boston
- Assignee: Wake Forest University Health Sciences,Genethon,Children's Medical Center Corporation
- Current Assignee: Wake Forest University Health Sciences,Genethon,Children's Medical Center Corporation
- Current Assignee Address: US NC Winston-Salem; FR Evry; US MA Boston
- Agency: Saul Ewing Arnstein & Lehr LLP
- Agent Kathryn Doyle
- Main IPC: A61K38/46
- IPC: A61K38/46 ; C12N9/16 ; A61K48/00 ; C12N7/00
Abstract:
The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
Public/Granted literature
- US10736945B2 Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM) Public/Granted day:2020-08-11
Information query
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