(Q42226636)

19 October 2017

title

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

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19 October 2017

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Laurent Monassier

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Nathalie Cartier

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object named as

Nathalie Cartier

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19 October 2017

Morgane Perdomini

1

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19 October 2017

Brahim Belbellaa

2

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19 October 2017

Laurence Reutenauer

4

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19 October 2017

Nadia Messaddeq

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19 October 2017

Ronald G Crystal

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19 October 2017

Patrick Aubourg

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19 October 2017

Hélène Puccio

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19 October 2017

language of work or name

English

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publication date

6 April 2014

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19 October 2017

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19 October 2017

volume

20

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19 October 2017

issue

5

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19 October 2017

page(s)

542-547

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https://scigraph.springernature.com/pub.10.1038/nm.3510

19 October 2017

exact match

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cites work

Molecular and functional alterations in a mouse cardiac model of Friedreich ataxia: activation of the integrated stress response, eIF2α phosphorylation, and the induction of downstream targets.

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12 December 2020

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

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12 December 2020

Mammalian frataxin controls sulfur production and iron entry during de novo Fe4S4 cluster assembly

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12 December 2020

Mammalian frataxin: an essential function for cellular viability through an interaction with a preformed ISCU/NFS1/ISD11 iron-sulfur assembly complex.

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12 December 2020

RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy

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12 December 2020

Cardiac transplantation: a temporary solution for Friedreich's ataxia-induced dilated cardiomyopathy

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12 December 2020

Iron and iron-responsive proteins in the cardiomyopathy of Friedreich's ataxia

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12 December 2020

A fatal mitochondrial disease is associated with defective NFU1 function in the maturation of a subset of mitochondrial Fe-S proteins

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12 December 2020

Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes

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12 December 2020

Cardiac energetics are abnormal in Friedreich ataxia patients in the absence of cardiac dysfunction and hypertrophy: an in vivo 31P magnetic resonance spectroscopy study.

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12 December 2020

Therapeutic developments in Friedreich ataxia

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12 December 2020

Molecular distinction between physiological and pathological cardiac hypertrophy: experimental findings and therapeutic strategies

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12 December 2020

Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features

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12 December 2020

Impaired myocardial perfusion reserve and fibrosis in Friedreich ataxia: a mitochondrial cardiomyopathy with metabolic syndrome

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12 December 2020

Friedreich's ataxia reveals a mechanism for coordinate regulation of oxidative metabolism via feedback inhibition of the SIRT3 deacetylase

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12 December 2020

The heart in Friedreich ataxia: definition of cardiomyopathy, disease severity, and correlation with neurological symptoms.

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12 December 2020

Fusion and fission: interlinked processes critical for mitochondrial health

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12 December 2020

Mortality in Friedreich ataxia

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12 December 2020

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

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12 December 2020

The in vivo mitochondrial two-step maturation of human frataxin.

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12 December 2020

Human frataxin is an allosteric switch that activates the Fe-S cluster biosynthetic complex

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12 December 2020

Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia

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12 December 2020

Mouse models for Friedreich ataxia exhibit cardiomyopathy, sensory nerve defect and Fe-S enzyme deficiency followed by intramitochondrial iron deposits

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12 December 2020

Friedreich ataxia: the oxidative stress paradox

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12 December 2020

Longitudinal strain in Friedreich Ataxia: a potential marker for early left ventricular dysfunction

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12 December 2020

Reciprocal regulation of microRNA-1 and insulin-like growth factor-1 signal transduction cascade in cardiac and skeletal muscle in physiological and pathological conditions

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12 December 2020

Calcium-calcineurin signaling in the regulation of cardiac hypertrophy

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12 December 2020

Normal left ventricular ejection fraction and mass but subclinical myocardial dysfunction in patients with Friedreich's ataxia.

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12 December 2020

A review of Friedreich ataxia clinical trial results

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12 December 2020

Intracellular calcium homeostasis in cardiac myocytes

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12 December 2020

Idebenone delays the onset of cardiac functional alteration without correction of Fe-S enzymes deficit in a mouse model for Friedreich ataxia

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12 December 2020

Clinical data and characterization of the liver conditional mouse model exclude neoplasia as a non-neurological manifestation associated with Friedreich's ataxia.

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12 December 2020

Frataxin deficiency causes upregulation of mitochondrial Lon and ClpP proteases and severe loss of mitochondrial Fe-S proteins

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12 December 2020

Mitochondrial impairment of human muscle in Friedreich ataxia in vivo

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12 December 2020

Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion

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12 December 2020

Persistent expression of biologically active anti-HER2 antibody by AAVrh.10-mediated gene transfer

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12 December 2020

Identifiers

DOI

10.1038/NM.3510

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19 October 2017

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