Invention Grant
- Patent Title: Methods for the treatment of Leber congenital amaurosis
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Application No.: US15267175Application Date: 2016-09-16
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Publication No.: US09777272B2Publication Date: 2017-10-03
- Inventor: Jean-Michel Rozet , Antoine Kichler , Isabelle Perrault , Josseline Kaplan , Xavier Gerard , Daniel Scherman , M. Arnold Munnich
- Applicant: Jean-Michel Rozet , Antoine Kichler , Isabelle Perrault , Josseline Kaplan , Xavier Gerard , Daniel Scherman , M. Arnold Munnich
- Applicant Address: FR Paris FR Paris FR Evry FR Paris FR Paris FR Evry FR Paris
- Assignee: 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
- Current Assignee: 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
- Current Assignee Address: FR Paris FR Paris FR Evry FR Paris FR Paris FR Evry FR Paris
- Agency: Whitham, Curtis & Cook, P.C.
- Priority: EP11305735 20110610
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N15/11 ; C07H21/02 ; C07H21/04 ; C12N15/113
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.
Public/Granted literature
- US20170044533A1 METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS Public/Granted day:2017-02-16
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