Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring 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.
Type:
Application
Filed:
September 16, 2016
Publication date:
February 16, 2017
Applicants:
INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP - Chimie ParisTech - Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
Inventors:
Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH
Abstract: The present invention relates to a method for treating a Leber congenital amaurosis in a patient harbouring 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.
Type:
Application
Filed:
November 18, 2014
Publication date:
April 23, 2015
Applicants:
INSERM (Institut National de la Sante et de la Recherche Medicale), CNRS (Centre National de la Recherche Scientifique), Genethon, Universite Paris Descartes, ENSCP - Chimie ParisTech - Ecole Nationale Superieure de Chimie de Paris, Universite d'Evry-Val-d'Essonne, Assistance Publique Hopitaux De Paris
Inventors:
Jean-Michel ROZET, Antoine KICHLER, Isabelle PERRAULT, Josseline KAPLAN, Xavier GERARD, Daniel SCHERMAN, M. Arnold MUNNICH