Loss-of-function of Endothelin receptor type A results in Oro-Oto-Cardiac syndrome.


Journal

American journal of medical genetics. Part A
ISSN: 1552-4833
Titre abrégé: Am J Med Genet A
Pays: United States
ID NLM: 101235741

Informations de publication

Date de publication:
05 2020
Historique:
received: 27 06 2019
revised: 06 02 2020
accepted: 06 02 2020
pubmed: 7 3 2020
medline: 13 1 2021
entrez: 6 3 2020
Statut: ppublish

Résumé

Craniofacial morphogenesis is regulated in part by signaling from the Endothelin receptor type A (EDNRA). Pathogenic variants in EDNRA signaling pathway components EDNRA, GNAI3, PCLB4, and EDN1 cause Mandibulofacial Dysostosis with Alopecia (MFDA), Auriculocondylar syndrome (ARCND) 1, 2, and 3, respectively. However, cardiovascular development is normal in MFDA and ARCND individuals, unlike Ednra knockout mice. One explanation may be that partial EDNRA signaling remains in MFDA and ARCND, as mice with reduced, but not absent, EDNRA signaling also lack a cardiovascular phenotype. Here we report an individual with craniofacial and cardiovascular malformations mimicking the Ednra

Identifiants

pubmed: 32133772
doi: 10.1002/ajmg.a.61531
pmc: PMC7202054
mid: NIHMS1573711
doi:

Substances chimiques

EDNRA protein, human 0
Receptor, Endothelin A 0
GNAI3 protein, human EC 3.6.5.1
GTP-Binding Protein alpha Subunits, Gi-Go EC 3.6.5.1

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

1104-1116

Subventions

Organisme : NIGMS NIH HHS
ID : F30 GM131672
Pays : United States
Organisme : NIDCR NIH HHS
ID : R01 DE023050
Pays : United States
Organisme : NIGMS NIH HHS
ID : R01 GM130142
Pays : United States

Informations de copyright

© 2020 Wiley Periodicals, Inc.

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Auteurs

Amanda Barone Pritchard (AB)

Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Stanley M Kanai (SM)

Department of Craniofacial Biology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA.

Bryan Krock (B)

Division of Genomic Diagnostics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Erica Schindewolf (E)

Center for Fetal Diagnosis and Treatment, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Jennifer Oliver-Krasinski (J)

Department of Pathology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Nahla Khalek (N)

Center for Fetal Diagnosis and Treatment, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Najeah Okashah (N)

Department of Pharmacology and Toxicology, Medical College of Georgia-Augusta University, Augusta, Georgia, USA.

Nevin A Lambert (NA)

Department of Pharmacology and Toxicology, Medical College of Georgia-Augusta University, Augusta, Georgia, USA.

Andre L P Tavares (ALP)

Department of Craniofacial Biology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA.

Elaine Zackai (E)

Division of Human Genetics, Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

David E Clouthier (DE)

Department of Craniofacial Biology, University of Colorado Anschutz Medical Campus, Aurora, Colorado, USA.

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Classifications MeSH