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Évry, France

French Institute of Health, Medical Research, French National Center for Scientific Research, Genethon, University of Paris Descartes, École Nationale Supérieure de Chimie de Paris, University of Évry Val d'Essonne and Assistance Publique Hopitaux De Paris | Date: 2014-11-18

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.2291+1655 A>G CEP290 mRNA

A pharmaceutical composition including at least one epigenome-modifying compound, for a use thereof in the treatment of genetic muscular diseases linked to a conformational disorder of at least one protein, said disorder causing the cellular degradation of the protein.

Genethon and French National Center for Scientific Research | Date: 2012-04-27

The invention concerns an adeno-associated viral vector comprising:

The present invention relates to methods for the production of biopharmaceuticals implementing a baculovirus-based system. These methods advantageously allow the production of biopharmaceuticals with a reduced number of or without contaminating baculoviral virions.

Genethon | Date: 2013-09-26

The invention relates to a method for the diagnosis, prognosis and therapeutic monitoring of muscular dystrophy, by detecting titin or one or more fragments of titin in a bodily fluid of a patient.

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