Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to energy failure
AssigneesInstitut National De La Sante Et De La Recherche Medicale (Inserm), Centre National de la Recherche Scientifique (CNRS), Universite De Strasbourg, Cornell University, Universite Paris-Saclay, Assistance Publique-HôPitaux De Paris (Aphp)
InventorsHélène Monique PUCCIO, Patrick AUBOURG, Ronald G. CRYSTAL, Pierre BOUGNERES
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Abstract
A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
Claims11
-12. (canceled)
An Adeno-Associated Virus (AAV) vector comprising a nucleic acid sequence encoding frataxin or a fragment thereof.
Dependencies are inferred from the claim text.