Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to energy failure

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).

1 . 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 reverse energy failure. 
     
     
         2 . The method according to  claim 1  wherein the cardiomyopathy due to energy failure is a cardiomyopathy associated with Friedreich ataxia and the gene that can reverse energy failure is a frataxin (FXN) encoding nucleic acid. 
     
     
         3 . The method according to  claim 2 , wherein said FXN encoding nucleic acid encodes for an amino acid sequence which is or includes SEQ ID NO:2. 
     
     
         4 . The method according to  claim 1  wherein the vector of claim comprises the nucleic acid sequence is or includes SEQ ID NO:1. 
     
     
         5 . The method according to  claim 1  wherein the vector is selected from the group consisting of adenovirus, retrovirus, herpesvirus and Adeno-Associated Virus (AAV) vectors. 
     
     
         6 . The method according to  claim 5  wherein the vector is an AAV vector. 
     
     
         7 . The method according to  claim 6  wherein the AAV vector is an AAV1, AAV2, AAV3, AAV4, AA5, AAV6, AAV7, AAV8, AAV9, AAVrh10 vector or any AAV derived vector. 
     
     
         8 . The method according to  claim 7  wherein the AAV vector is an AAVrh10 vector. 
     
     
         9 . The method according to  claim 1  wherein the vector is administered intracoronary or directly into the myocardium of the subject. 
     
     
         10 . The method according to  claim 1  wherein the vector is administered by intravenous injection. 
     
     
         11 . A pharmaceutical composition for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can reverse energy failure. 
     
     
         12 . The pharmaceutical composition according to  claim 11  wherein the cardiomyopathy due to energy failure is a cardiomyopathy associated with Friedreich ataxia and the gene that can reverse energy failure is the frataxin (FXN) encoding nucleic acid.