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Mutations in SLC39A14 disrupt manganese homeostasis and cause childhood-onset parkinsonism-dystonia.

Published version
Peer-reviewed

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Authors

Tuschl, Karin 
Meyer, Esther 
Valdivia, Leonardo E 
Zhao, Ningning 
Dadswell, Chris 

Abstract

Although manganese is an essential trace metal, little is known about its transport and homeostatic regulation. Here we have identified a cohort of patients with a novel autosomal recessive manganese transporter defect caused by mutations in SLC39A14. Excessive accumulation of manganese in these patients results in rapidly progressive childhood-onset parkinsonism-dystonia with distinctive brain magnetic resonance imaging appearances and neurodegenerative features on post-mortem examination. We show that mutations in SLC39A14 impair manganese transport in vitro and lead to manganese dyshomeostasis and altered locomotor activity in zebrafish with CRISPR-induced slc39a14 null mutations. Chelation with disodium calcium edetate lowers blood manganese levels in patients and can lead to striking clinical improvement. Our results demonstrate that SLC39A14 functions as a pivotal manganese transporter in vertebrates.

Description

Keywords

Adolescent, Animals, Cation Transport Proteins, Child, Child, Preschool, Dystonic Disorders, Female, Genetic Predisposition to Disease, HEK293 Cells, Homeostasis, Humans, Male, Manganese, Mutation, Parkinsonian Disorders, Pedigree, Young Adult, Zebrafish

Journal Title

Nat Commun

Conference Name

Journal ISSN

2041-1723
2041-1723

Volume Title

7

Publisher

Springer Science and Business Media LLC
Sponsorship
Action Medical Research