The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Mar. 17, 2020

Filed:

Jul. 31, 2015
Applicants:

Association Institut DE Myologie, Paris, FR;

Institut National DE LA Sante ET DE LA Recherche Medicale, Paris, FR;

Universite Pierre ET Marie Curie (Paris 6), Paris, FR;

Inventors:

Martine Barkats, Charenton le Pont, FR;

Maria-Grazia Biferi, Paris, FR;

Thomas Voit, London, GB;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C07H 21/02 (2006.01); A61K 48/00 (2006.01); C12N 15/113 (2010.01); A61K 31/713 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/1137 (2013.01); A61K 31/713 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12Y 115/01001 (2013.01); C12N 2310/11 (2013.01); C12N 2320/32 (2013.01); C12N 2320/33 (2013.01); C12N 2330/51 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14343 (2013.01); C12N 2799/025 (2013.01);
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.


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