Invention Grant
- Patent Title: Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
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Application No.: US15384976Application Date: 2016-12-20
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Publication No.: US09895426B2Publication Date: 2018-02-20
- 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: A61K48/00
- IPC: A61K48/00 ; A61K38/46 ; C12N7/00 ; C07H21/00 ; C07H21/04 ; C12N15/00 ; C12N15/85 ; C12P21/06
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
- US20170112905A1 SYSTEMIC GENE REPLACEMENT THERAPY FOR TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY (XLMTM) Public/Granted day:2017-04-27
Information query
