Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
AssigneesInstitut National De La Sante Et De La Recherche Medicale (Inserm), Cnrs (Centre National De La Recherche Scientifique), 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
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).
Claims21
A pharmaceutical composition for treating cardiomyopathy in a Friedreich ataxia patient exhibiting cardiomyopathy, the composition comprising an Adeno-Associated Virus (AAV) vector which comprises a heterologous sequence which consists of a coding sequence encoding FXN or a fragment thereof operably linked to regulatory sequences wherein the fragment comprises a coding sequence encoding amino acids 81-210 of SEQ ID NO:2, wherein said AAV vector is formulated for delivery into myocardium in said patient.
A method for treating cardiomyopathy in a Friedreich ataxia patient exhibiting cardiomyopathy, comprising directly administering into the heart of said patient a therapeutically effective amount of an AAV vector which comprises a heterologous sequence which consists of a coding sequence encoding frataxin (FXN) or a fragment thereof operably linked to regulatory sequences wherein the fragment comprises a coding sequence encoding amino acids 81-210 of SEQ ID NO:2.
Dependencies are inferred from the claim text.