Orthod Craniofac Res. 2019;22(Suppl. 1):163–167. wileyonlinelibrary.com/journal/ocr
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163 © 2019 John Wiley & Sons A/S.
Published by John Wiley & Sons Ltd
1 | INTRODUCTION
Craniofacial development is a highly coordinated process under a
tight genetic and epigenetic control. Any mishaps during the early
developmental process will result in a range of craniofacial malfor-
mations.
1
Craniofacial microsomia (CFM) is a congenital disorder with
unilateral or bilateral defects in the derivatives of the first and sec-
ond branchial arches. The phenotypic presentation of these patients
includes defects of the external ear, middle ear, temporomandibular
joint, mandible, muscles of facial expression and mastication leading to
Received: 29 November 2018
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Revised: 19 December 2018
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Accepted: 4 December 2018
DOI: 10.1111/ocr.12259
SUPPLEMENT ARTICLE
A novel nonsense substitution identified in the AMIGO2 gene in
an Occulo-Auriculo-Vertebral spectrum patient
Shankar Rengasamy Venugopalan
1
| Emily Farrow
2
| Pedro A. Sanchez–Lara
3,4
|
Stephen Yen
4
| Michael Lypka
2
| Shao Jiang
2
| Veerasathpurush Allareddy
5
Shankar Rengasamy Venugopalan and Emily Farrow these authors contributed equally.
1
School of Dentistry, University of Missouri
Kansas City, Kansas City, Missouri
2
Children’s Mercy Hospitals, Kansas City,
Missouri
3
Cedars–Sinai Medical Center, Los Angeles,
California
4
Children’s Hospital Los Angeles, Los
Angeles, California
5
College of Dentistry, University of Illinois at
Chicago, Chicago, Illinois
Correspondence
Shankar Rengasamy Venugopalan,
Department of Orthodontics and
Dentofacial Orthopedics, School of
Dentistry University of Missouri Kansas City,
Kansas City, MO.
Email: rengavs@umkc.edu
Funding information
American Association of Orthodontist
Foundation; University of Missouri Kansas
City Center for Excellence in the Study of
Dental and Musculoskeletal Tissues
Structured Abstract
Objective: Craniofacial microsmia is the second most common congenital disorder
with mostly unilateral defects of ear, temporomandibular joint, mandible, and mus-
cles of facial expression and mastication. The objective of this study was to identify,
if there were any, de novo germline or somatic variants in a patient with Occulo-
Auriculo-Vertebral Spectrum (OAVS) using whole-exome sequencing.
Settings and Sample Population: Trio/Family-based study of an OAVS proband.
Materials and Methods: Children’s Mercy Hospital Institutional Review Board ap-
proved this study and a request-to-rely was procured from the University of Missouri
Kansas City IRB. Informed assent/consent was obtained for all family members prior
to any research activities. The peripheral blood/affected side tissues from corrective
surgery of the proband and peripheral blood samples from unaffected parents were
collected. The isolated genomic DNA were enriched for exomes and sequenced on
an Illlumina HiSeq 2500 instrument yielding paired-end 125 nucleotide reads (84X
coverage). Gapped alignment to reference sequences (GRCh37.p5) was performed
with BWA and the GATK and analysis completed using custom-developed software.
Results: Analyses revealed that the proband carried a de novo germ line nonsense
substitution (c.901C>T) in AMIGO2 gene, and missense substitutions in ZCCHC14
(c.1198C>T), and in SZT2 genes (c.2951C>T).
Conclusions: The nonsense substitution in AMIGO2 gene introduces a premature
stop codon possibly rendering the gene non-functional via nonsense-mediated path-
way decay—therefore considered a stronger candidate. Further functional studies
are required to confirm whether loss-of-function variants in AMIGO2 can cause
OAVS.
KEYWORDS
AMIGO2, craniofacial microsomia, Goldenhar Syndrome, Occulo-Auriculo-Vertebral Spectrum