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:
Nov. 08, 2016

Filed:

Nov. 18, 2014
Applicants:

Jean-michel Rozet, Paris, FR;

Antoine Kichler, Evry, FR;

Isabelle Perrault, Paris, FR;

Josseline Kaplan, Paris, FR;

Xavier Gerard, Paris, FR;

Daniel Scherman, Paris, FR;

M. Arnold Munnich, Paris, FR;

Inventors:

Jean-Michel Rozet, Paris, FR;

Antoine Kichler, Evry, FR;

Isabelle Perrault, Paris, FR;

Josseline Kaplan, Paris, FR;

Xavier Gerard, Paris, FR;

Daniel Scherman, Paris, FR;

M. Arnold Munnich, Paris, FR;

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/02 (2006.01); C07H 21/04 (2006.01); A61K 48/00 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 48/005 (2013.01); C12N 2310/11 (2013.01); C12N 2320/33 (2013.01); C12N 2320/34 (2013.01);
Abstract

The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to said patient at least one antisense oligonucleotide complementary to nucleic acid sequence that is necessary for preventing splicing of the cryptic exon inserted into the mutant c.2291+1655 A>G CEP290 mRNA.


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