Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
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United States of America Patent
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Jun 30, 2015
Grant Date -
Aug 7, 2014
app pub date -
Feb 1, 2013
filing date -
Feb 1, 2013
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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).
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