Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to Friedreich ataxia
AssigneesAphp (Assistance Publique - HôPitaux De Paris), Cornell University, Institut National De La Sante Et De La Recherche M, Centre National de la Recherche Scientifique (CNRS), Universite De Strasbourg
InventorsHelene 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).
Claims6
A method for treating cardiomyopathy in a subject with Friedreich ataxia in need thereof, the method comprising administering by intravenous injection to said subject a therapeutically effective amount of a vector which comprises nucleic acid encoding a frataxin (FXN) operably linked to regulatory sequences, wherein the vector is an AAVrh10 vector.
Dependencies are inferred from the claim text.