Invention Grant
- Patent Title: Treatment of amyotrophic lateral sclerosis
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Application No.: US15327011Application Date: 2015-07-31
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Publication No.: US10590420B2Publication Date: 2020-03-17
- Inventor: Martine Barkats , Maria-Grazia Biferi , Thomas Voit
- Applicant: ASSOCIATION INSTITUT DE MYOLOGIE , INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE , UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
- Applicant Address: FR Paris FR Paris FR Paris
- Assignee: ASSOCIATION INSTITUT DE MYOLOGIE,INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE,UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
- Current Assignee: ASSOCIATION INSTITUT DE MYOLOGIE,INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE,UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
- Current Assignee Address: FR Paris FR Paris FR Paris
- Agency: Arrigo, Lee, Guttman & Mouta-Bellum LLP
- Priority: EP14306228 20140731
- International Application: PCT/EP2015/067722 WO 20150731
- International Announcement: WO2016/016449 WO 20160204
- Main IPC: C07H21/04
- IPC: C07H21/04 ; C07H21/02 ; A61K48/00 ; C12N15/113 ; A61K31/713 ; C12N7/00 ; C12N15/86

Abstract:
The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.
Public/Granted literature
- US20170152517A1 TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS Public/Granted day:2017-06-01
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