Large duplication in LMBR1 gene in a large Chinese pedigree with triphalangeal thumb polysyndactyly syndrome

Am J Med Genet A. 2020 Sep;182(9):2117-2123. doi: 10.1002/ajmg.a.61757. Epub 2020 Jul 14.

Abstract

Polydactyly and syndactyly are digital abnormalities in limb-associated birth defects usually caused by genetic disorders. In this study, a five-generation Chinese pedigree was found with triphalangeal thumb polysyndactyly syndrome (TPTPS), showing an autosomal dominant pattern of inheritance. We utilized linkage analysis and whole genome sequencing (WGS) for the genetic diagnosis of this pedigree. Linkage analysis was performed using a genome-wide single nucleotide polymorphism (SNP) chip and three genomic regions were identified in chromosomes 2, 6, and 7 with significant linkage signals. WGS discovered a copy number variation (CNV) mutation caused by a large duplication region at the tail of chromosome 7 located in exons 1-5 of the LMBR1 gene, including the zone of polarizing activity regulatory sequence (ZRS), with a length of approximately 180 kb. A real-time polymerase chain reaction (PCR) assay confirmed the duplication. The findings of our study supported the notion that large duplications including the ZRS caused TPTPS. Our study showed that linkage analysis in combination with WGS could successfully identify the disease locus and causative mutation in TPTPS, which could help elucidate the molecular mechanisms and genotype-phenotype correlations in polydactyly.

Keywords: LMBR1; copy number variations; linkage analysis; polydactyly; whole genome sequencing.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Congenital Abnormalities / genetics*
  • Congenital Abnormalities / physiopathology
  • DNA Copy Number Variations / genetics
  • Female
  • Genetic Linkage / genetics
  • Genetic Predisposition to Disease*
  • Humans
  • Limb Deformities, Congenital / genetics*
  • Limb Deformities, Congenital / pathology
  • Male
  • Mandibulofacial Dysostosis / genetics*
  • Mandibulofacial Dysostosis / physiopathology
  • Membrane Proteins / genetics*
  • Mutation / genetics
  • Pedigree
  • Polymorphism, Single Nucleotide / genetics
  • Whole Genome Sequencing
  • Young Adult

Substances

  • LMBR1 protein, human
  • Membrane Proteins

Supplementary concepts

  • Patterson Stevenson syndrome