Mitochondrial functions and rare diseases.


Journal

Molecular aspects of medicine
ISSN: 1872-9452
Titre abrégé: Mol Aspects Med
Pays: England
ID NLM: 7603128

Informations de publication

Date de publication:
02 2020
Historique:
received: 03 10 2019
revised: 26 12 2019
accepted: 27 12 2019
pubmed: 8 2 2020
medline: 9 2 2021
entrez: 8 2 2020
Statut: ppublish

Résumé

Mitochondria are dynamic cellular organelles responsible for a large variety of biochemical processes as energy transduction, REDOX signaling, the biosynthesis of hormones and vitamins, inflammation or cell death execution. Cell biology studies established that 1158 human genes encode proteins localized to mitochondria, as registered in MITOCARTA. Clinical studies showed that a large number of these mitochondrial proteins can be altered in expression and function through genetic, epigenetic or biochemical mechanisms including the interaction with environmental toxics or iatrogenic medicine. As a result, pathogenic mitochondrial genetic and functional defects participate to the onset and the progression of a growing number of rare diseases. In this review we provide an exhaustive survey of the biochemical, genetic and clinical studies that demonstrated the implication of mitochondrial dysfunction in human rare diseases. We discuss the striking diversity of the symptoms caused by mitochondrial dysfunction and the strategies proposed for mitochondrial therapy, including a survey of ongoing clinical trials.

Identifiants

pubmed: 32029308
pii: S0098-2997(19)30117-7
doi: 10.1016/j.mam.2019.100842
pii:
doi:

Substances chimiques

Mitochondrial Proteins 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

100842

Informations de copyright

Copyright © 2020 Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors declare no conflict of interest.

Auteurs

L Dard (L)

Bordeaux University, 33000, Bordeaux, France; INSERM U1211, 33000, Bordeaux, France; CELLOMET, CGFB-146 Rue Léo Saignat, Bordeaux, France.

W Blanchard (W)

Bordeaux University, 33000, Bordeaux, France; INSERM U1211, 33000, Bordeaux, France; CELLOMET, CGFB-146 Rue Léo Saignat, Bordeaux, France.

C Hubert (C)

Bordeaux University, 33000, Bordeaux, France; INSERM U1211, 33000, Bordeaux, France.

D Lacombe (D)

Bordeaux University, 33000, Bordeaux, France; INSERM U1211, 33000, Bordeaux, France; CHU de Bordeaux, Service de Génétique Médicale, F-33076, Bordeaux, France.

R Rossignol (R)

Bordeaux University, 33000, Bordeaux, France; INSERM U1211, 33000, Bordeaux, France; CELLOMET, CGFB-146 Rue Léo Saignat, Bordeaux, France. Electronic address: rodrigue.rossignol@u-bordeaux.fr.

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Classifications MeSH