Patents Assigned to INSERM (Institut National de la Sante et de la Racherche Medicale)
  • METHODS FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
    Publication number: 20140179767
    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