US 2021/0340565 A1 · METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE

Methods and pharmaceutical composition 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-Sud Xi, Assistance Publique-HôPitaux De Paris (Aphp)

InventorsHelene Monique PUCCIO, Patrick AUBOURG, Ronald G. CRYSTAL, Pierre BOUGNERES

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Abstract

The present invention relates to a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.

Claims20

Dependent claims

A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure.

Dependencies are inferred from the claim text.