Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia-Reference-Cited by-同舟云学术

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

Published:2014-04-06 Issue:5 Volume:20 Page:542-547
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Perdomini Morgane,Belbellaa Brahim,Monassier Laurent,Reutenauer Laurence,Messaddeq Nadia,Cartier Nathalie,Crystal Ronald G,Aubourg Patrick,Puccio Hélène

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/nm.3510.pdf

Reference37 articles.

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3. Weidemann, F. et al. The heart in Friedreich ataxia: definition of cardiomyopathy, disease severity, and correlation with neurological symptoms. Circulation 125, 1626–1634 (2012).

4. Campuzano, V. et al. Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes. Hum. Mol. Genet. 6, 1771–1780 (1997).

5. Campuzano, V. et al. Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion. Science 271, 1423–1427 (1996).

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