Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia. Morgane Perdomini, Brahim Belbellaa, Laurent Monassier, Laurence Reutenauer, Nadia Messaddeq, Nathalie Cartier, Ronald G Crystal, Patrick Aubourg & Hélène Puccio; Nature Medicine (2014) doi:10.1038/nm.3510

KEYWORDS: Cardiac failure, conditional mouse model, adeno-associated virus rh10 vector, establish the preclinical proof of concept, FRDA cardiomyopathy.