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

被引：0

作者：

Morgane Perdomini

Brahim Belbellaa

Laurent Monassier

Laurence Reutenauer

Nadia Messaddeq

Nathalie Cartier

Ronald G Crystal

Patrick Aubourg

Hélène Puccio

机构：

[1] Départment de Médecine Translationnelle et Neurogénétique,Department of Genetic Medicine

[2] Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC),undefined

[3] INSERM,undefined

[4] U596,undefined

[5] CNRS,undefined

[6] UMR7104,undefined

[7] Université de Strasbourg,undefined

[8] Collège de France,undefined

[9] Chaire de génétique humaine,undefined

[10] Laboratoire de Neurobiologie et Pharmacologie Cardiovasculaire,undefined

[11] Fédération de Médecine Translationnelle,undefined

[12] Faculté de Médecine,undefined

[13] Université de Strasbourg,undefined

[14] INSERM,undefined

[15] U986,undefined

[16] Weill Cornell Medical College,undefined

[17] University Paris-Sud,undefined

[18] Assistance Publique-Hôpitaux de Paris,undefined

来源：

Nature Medicine | 2014年 / 20卷

关键词：

D O I：

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中图分类号：

学科分类号：

摘要：

In Friedreich's ataxia, caused by mutation of the gene encoding the mitochondrial protein frataxin, the major cause of mortality is heart failure. Using mice lacking frataxin in the heart, Hélène Puccio and her colleagues demonstrate that frataxin gene therapy can correct mitochondrial metabolism and reverse heart damage, raising the possibility of a gene therapy treatment for this disease.

引用

页码：542 / 547

页数：5

共 50 条

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