Blepharospasm: A genetic screening study in 132 patients.


Journal

Parkinsonism & related disorders
ISSN: 1873-5126
Titre abrégé: Parkinsonism Relat Disord
Pays: England
ID NLM: 9513583

Informations de publication

Date de publication:
07 2019
Historique:
received: 03 01 2019
revised: 28 03 2019
accepted: 01 04 2019
pubmed: 9 4 2019
medline: 22 5 2020
entrez: 9 4 2019
Statut: ppublish

Résumé

Blepharospasm is a common type of focal dystonia that involves involuntary eyelid spasms and eye closure. In familial cases, an autosomal dominant pattern of inheritance is noted with reduced penetrance. Few genes have been associated with the disease including GNAL and CIZ1. A whole exome sequencing study published lately suggested TOR2A and REEP4 as potential candidate genes. Sanger sequencing of GNAL, CIZ1, TOR2A and REEP4 exons including exon-intron boundaries in 132 patients diagnosed primarily with blepharospasm and/or Meige's syndrome. All variants detected in GNAL, CIZ1 and TOR2A seem to be benign. Sequencing of REEP4 revealed the presence of two nonsynonymous SNVs, one potential splice site variant and one indel all predicted to be damaging by in silico algorithms. Sequencing REEP4 in larger blepharospasm cohorts and functional studies will need to be performed to further elucidate the association between REEP4 and the disease.

Identifiants

pubmed: 30956059
pii: S1353-8020(19)30197-X
doi: 10.1016/j.parkreldis.2019.04.003
pmc: PMC6739171
mid: NIHMS1526706
pii:
doi:

Substances chimiques

Membrane Transport Proteins 0
REEP4 protein, human 0

Types de publication

Journal Article Research Support, N.I.H., Intramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

315-318

Subventions

Organisme : Intramural NIH HHS
ID : Z01 AG000957
Pays : United States
Organisme : Intramural NIH HHS
ID : Z01 AG000957-05
Pays : United States

Informations de copyright

Copyright © 2019 Elsevier Ltd. All rights reserved.

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Auteurs

Monia Hammer (M)

Laboratory of Neurogenetics, National Institute on Aging, Bethesda, MD, 20892, USA. Electronic address: hammermb@mail.nih.gov.

Alexandra Abravanel (A)

Laboratory of Neurogenetics, National Institute on Aging, Bethesda, MD, 20892, USA.

Elizabeth Peckham (E)

Human Motor Control Section, National Institute of Neurological Disorders and Stroke, Bethesda, MD, 20892, USA.

Ava Mahloogi (A)

Laboratory of Neurogenetics, National Institute on Aging, Bethesda, MD, 20892, USA.

Elisa Majounie (E)

Laboratory of Neurogenetics, National Institute on Aging, Bethesda, MD, 20892, USA.

Mark Hallett (M)

Human Motor Control Section, National Institute of Neurological Disorders and Stroke, Bethesda, MD, 20892, USA.

Andrew Singleton (A)

Laboratory of Neurogenetics, National Institute on Aging, Bethesda, MD, 20892, USA.

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Classifications MeSH