POLG mutations presenting as Charcot-Marie-Tooth disease.


Journal

Journal of the peripheral nervous system : JPNS
ISSN: 1529-8027
Titre abrégé: J Peripher Nerv Syst
Pays: United States
ID NLM: 9704532

Informations de publication

Date de publication:
06 2019
Historique:
received: 15 02 2019
revised: 27 02 2019
accepted: 02 03 2019
pubmed: 8 3 2019
medline: 19 5 2020
entrez: 8 3 2019
Statut: ppublish

Résumé

We report on two patients, with different POLG mutations, in whom axonal neuropathy dominated the clinical picture. One patient presented with late onset sensory axonal neuropathy caused by a homozygous c.2243G>C (p.Trp748Ser) mutation that resulted from uniparental disomy of the long arm of chromosome 15. The other patient had a complex phenotype that included early onset axonal Charcot-Marie-Tooth disease (CMT) caused by compound heterozygous c.926G>A (p.Arg309His) and c.2209G>C (p.Gly737Arg) mutations.

Identifiants

pubmed: 30843307
doi: 10.1111/jns.12313
pmc: PMC8287532
mid: NIHMS1700226
doi:

Substances chimiques

DNA Polymerase gamma EC 2.7.7.7
POLG protein, human EC 2.7.7.7

Types de publication

Case Reports Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

213-218

Subventions

Organisme : NINDS NIH HHS
ID : U54 NS065712
Pays : United States

Informations de copyright

© 2019 Peripheral Nerve Society.

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Auteurs

Jade Phillips (J)

Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Steve Courel (S)

Department of Human Genetics and Hussman Institute for Human Genomics, University of Miami, Miami, Florida.

Adriana P Rebelo (AP)

Department of Human Genetics and Hussman Institute for Human Genomics, University of Miami, Miami, Florida.

Dana M Bis-Brewer (DM)

Department of Human Genetics and Hussman Institute for Human Genomics, University of Miami, Miami, Florida.

Tanya Bardakjian (T)

Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Lois Dankwa (L)

Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Ali G Hamedani (AG)

Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

Stephan Züchner (S)

Department of Human Genetics and Hussman Institute for Human Genomics, University of Miami, Miami, Florida.

Steven S Scherer (SS)

Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.

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Classifications MeSH