Genome-wide association studies in dogs and humans identify ADAMTS20 as a risk variant for cleft lip and palate

PLoS Genet. 2015 Mar 23;11(3):e1005059. doi: 10.1371/journal.pgen.1005059. eCollection 2015 Mar.

Abstract

Cleft lip with or without cleft palate (CL/P) is the most commonly occurring craniofacial birth defect. We provide insight into the genetic etiology of this birth defect by performing genome-wide association studies in two species: dogs and humans. In the dog, a genome-wide association study of 7 CL/P cases and 112 controls from the Nova Scotia Duck Tolling Retriever (NSDTR) breed identified a significantly associated region on canine chromosome 27 (unadjusted p=1.1 x 10(-13); adjusted p= 2.2 x 10(-3)). Further analysis in NSDTR families and additional full sibling cases identified a 1.44 Mb homozygous haplotype (chromosome 27: 9.29 - 10.73 Mb) segregating with a more complex phenotype of cleft lip, cleft palate, and syndactyly (CLPS) in 13 cases. Whole-genome sequencing of 3 CLPS cases and 4 controls at 15X coverage led to the discovery of a frameshift mutation within ADAMTS20 (c.1360_1361delAA (p.Lys453Ilefs*3)), which segregated concordant with the phenotype. In a parallel study in humans, a family-based association analysis (DFAM) of 125 CL/P cases, 420 unaffected relatives, and 392 controls from a Guatemalan cohort, identified a suggestive association (rs10785430; p =2.67 x 10-6) with the same gene, ADAMTS20. Sequencing of cases from the Guatemalan cohort was unable to identify a causative mutation within the coding region of ADAMTS20, but four coding variants were found in additional cases of CL/P. In summary, this study provides genetic evidence for a role of ADAMTS20 in CL/P development in dogs and as a candidate gene for CL/P development in humans.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • ADAM Proteins / genetics*
  • ADAMTS Proteins
  • Animals
  • Brain / abnormalities*
  • Brain / pathology
  • Cleft Lip / genetics*
  • Cleft Lip / pathology
  • Cleft Palate / genetics*
  • Cleft Palate / pathology
  • Dogs
  • Frameshift Mutation
  • Genome-Wide Association Study*
  • Haplotypes
  • Humans
  • Polymorphism, Single Nucleotide

Substances

  • ADAM Proteins
  • ADAMTS Proteins
  • ADAMTS20 protein, human

Supplementary concepts

  • Orofacial Cleft 1

Associated data

  • dbGaP/PHS000440.V1.P1
  • Dryad/10.5061/dryad.J8R8Q