METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY ASSOCIATED WITH FRIEDREICH ATAXIA

Abstract: the present disclosure provides methods and compositions for the treatment of cardiomyopathy associated with friedreich ataxia. the methods and compositions of the present disclosure comprise aav vectors and aav viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a frataxin polypeptide.

Inventor(s): Ronald G. CRYSTAL, Stephen M. KAMINSKY, Dolan SONDHI, Jay A. BARTH, Richie KHANNA

CPC Classification: A61K48/0033 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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