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. 2991+1655 A>G CEP290 mRNA.
Type:
Grant
Filed:
March 3, 2020
Date of Patent:
November 8, 2022
Assignees:
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), GENETHON, UNIVERSITE PARIS DESCARTES, ENSCP—CHIMIE PARISTECH—ECOLE NATIONALE SUPERIEURE DE CHIME DE PARIS, ASSISTANCE PUBLIQUE—HOPITAUX DE PARIS
Inventors:
Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman, M. Arnold Munnich