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Altered 2-thiouridylation impairs mitochondrial translation in reversible infantile respiratory chain deficiency.

Published version
Peer-reviewed

Type

Article

Change log

Authors

Boczonadi, Veronika 
Smith, Paul M 
Pyle, Angela 
Gomez-Duran, Aurora 
Schara, Ulrike 

Abstract

Childhood-onset mitochondrial encephalomyopathies are severe, relentlessly progressive conditions. However, reversible infantile respiratory chain deficiency (RIRCD), due to a homoplasmic mt-tRNA(Glu) mutation, and reversible infantile hepatopathy, due to tRNA 5-methylaminomethyl-2-thiouridylate methyltransferase (TRMU) deficiency, stand out by showing spontaneous recovery, and provide the key to treatments of potential broader relevance. Modification of mt-tRNA(Glu) is a possible functional link between these two conditions, since TRMU is responsible for 2-thiouridylation of mt-tRNA(Glu), mt-tRNA(Lys) and mt-tRNA(Gln). Here we show that down-regulation of TRMU in RIRCD impairs 2-thiouridylation and exacerbates the effect of the mt-tRNA(Glu) mutation by triggering a mitochondrial translation defect in vitro. Skeletal muscle of RIRCD patients in the symptomatic phase showed significantly reduced 2-thiouridylation. Supplementation with l-cysteine, which is required for optimal TRMU function, rescued respiratory chain enzyme activities in human cell lines of patients with RIRCD as well as deficient TRMU. Our results show that l-cysteine supplementation is a potential treatment for RIRCD and for TRMU deficiency, and is likely to have broader application for the growing group of intra-mitochondrial translation disorders.

Description

Keywords

Cell Line, Cysteine, Gene Expression Regulation, Humans, Mitochondria, Mitochondrial Diseases, Mitochondrial Encephalomyopathies, Mitochondrial Proteins, Muscle, Skeletal, Mutation, Myoblasts, Oxidative Phosphorylation, Protein Biosynthesis, RNA, Transfer, Thiouridine, tRNA Methyltransferases

Journal Title

Hum Mol Genet

Conference Name

Journal ISSN

0964-6906
1460-2083

Volume Title

22

Publisher

Oxford University Press (OUP)
Sponsorship
Wellcome Trust (101876/Z/13/Z)