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:
Nov. 19, 2013
Filed:
Oct. 18, 2010
Nicolas Levy, Marseille Cedex, FR;
Martin Krahn, Marseille Cedex, FR;
Marc Bartoli, Marseille Cedex, FR;
Luis Garcia, Paris Cedex, FR;
Nicolas Levy, Marseille Cedex, FR;
Martin Krahn, Marseille Cedex, FR;
Marc Bartoli, Marseille Cedex, FR;
Luis Garcia, Paris Cedex, FR;
Institut National de la Sante et de la Recherche Medicale (INSERM), Paris, FR;
Universite de la Mediterranee—Aix Marseille II, Marseilles, FR;
Abstract
The present invention relates to methods for restoring the function of a mutated dysferlin comprising the step of preventing splicing of one or more exons which encode amino acid sequences that cause said dysferlin dysfunction. Particularly, the splicing of exon 32 is prevented. The present invention also relates to a method for treating a dysferlinopathy in a patient in need thereof, comprising the step of administering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of one or more exons which encode amino acid sequences that cause said dysfunction. Particularly, the splicing of exon 32 is prevented.