Invention Grant
- Patent Title: Modified U7 snRNAs for treatment of neuromuscular diseases
- Patent Title (中): 修饰的U7 snRNA用于治疗神经肌肉疾病
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Application No.: US13635356Application Date: 2011-03-17
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Publication No.: US09080170B2Publication Date: 2015-07-14
- Inventor: Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
- Applicant: Luis Garcia , Denis Furling , Cyriaque Beley , Thomas Voit
- Applicant Address: FR Paris
- Assignee: ASSOCIATION INSTITUT DE MYOLOGIE
- Current Assignee: ASSOCIATION INSTITUT DE MYOLOGIE
- Current Assignee Address: FR Paris
- Agency: Miles & Stockbridge P.C.
- International Application: PCT/EP2011/054026 WO 20110317
- International Announcement: WO2011/113889 WO 20110922
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C12N15/11
Abstract:
The present invention relates to a method to improve the activity of engineered U7 snRNAs used in the context of RNA-based therapeutics; particularly in exon skipping, exon inclusion, and mRNA eradication strategies. The resulting modified U7 snRNAs are useful for treating neuromuscular diseases, in particular Duchenne neuromuscular dystrophy, myotonic dystrophy DM1 and spinal muscular atrophy.
Public/Granted literature
- US20130045538A1 MODIFIED U7 SNRNAS FOR TREATMENT OF NEUROMUSCULAR DISEASES Public/Granted day:2013-02-21
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