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:
June 8, 2012
Publication date:
June 26, 2014
Applicants:
INSERM (Institut National de la Sante et de la Racherche Medicale), GENETHON, CNRS (Centre National de la Racherche Scientifique
Inventors:
Jean-Michel Rozet, Antoine Kichler, Isabelle Perrault, Josseline Kaplan, Xavier Gerard, Daniel Scherman
Abstract: The present application relates to oligonucleotides which inhibit expression of the OB-RGRP protein and to uses thereof for preventing and/or treating leptin-related pathological conditions. It also relates to a method for detecting compounds which modify the interaction between proteins of the OB-RGRP family and the leptin receptor. This detection may be carried out by measuring the energy transfer between fusion proteins composed of these proteins and of energy-donor and -acceptor proteins.
Type:
Application
Filed:
February 9, 2004
Publication date:
January 13, 2005
Applicants:
Aventis Pharma S.A., Institut National De La Sante Et De La Racherche Medicale, Institut National De La Centre National De La Rechercher Scientifique
Inventors:
Ralf Jockers, Cyril Couturier, Eugen Uhlmann