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.
Patent No.:
Date of Patent:
Jul. 14, 2015
Filed:
Mar. 17, 2011
Applicants:
Luis Garcia, Bailly, FR;
Denis Furling, Champigny-sur-Marne, FR;
Cyriaque Beley, Fontenay le Fleury, FR;
Thomas Voit, Boullay-les Troux, FR;
Inventors:
Luis Garcia, Bailly, FR;
Denis Furling, Champigny-sur-Marne, FR;
Cyriaque Beley, Fontenay le Fleury, FR;
Thomas Voit, Boullay-les Troux, FR;
Assignee:
ASSOCIATION INSTITUT DE MYOLOGIE, Paris, FR;
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C12N 15/11 (2006.01);
U.S. Cl.
CPC ...
C12N 15/111 (2013.01); C12N 2310/11 (2013.01); C12N 2320/33 (2013.01); C12N 2320/34 (2013.01); C12N 2330/51 (2013.01);
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.