Novel copy number variants and major limb reduction malformation: Report of three cases

Am J Med Genet A. 2016 May;170A(5):1245-50. doi: 10.1002/ajmg.a.37550. Epub 2016 Jan 8.

Abstract

Limb reduction malformations are highly heterogeneous in their clinical presentation and so, predicting the underlying mutation on a clinical basis can be challenging. Molecular karyotyping is a powerful genomic tool that has quickly become the mainstay for the study of children with malformation syndromes. We describe three patients with major limb reduction anomalies in whom pathogenic copy number variants were identified on molecular karyotyping. These include a patient with hypoplastic phalanges and absent hallux bilaterally with de novo deletion of 11.9 Mb on 7p21.1-22.1 spanning 63 genes including RAC1, another patient with severe Holt-Oram syndrome and a large de novo deletion 2.2 Mb on 12q24.13-24.21 spanning 20 genes including TBX3 and TBX5, and a third patient with acheiropodia who had a nullizygous deletion of 102 kb on 7q36.3 spanning LMBR1. We discuss the potential of these novel genomic rearrangements to improve our understanding of limb development in humans.

Keywords: Adams-Oliver syndrome; CGH; LMBR1; RAC1; TBX3; TBX5.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child, Preschool
  • DNA Copy Number Variations / genetics
  • Ectodermal Dysplasia / genetics*
  • Ectodermal Dysplasia / physiopathology
  • Humans
  • Infant
  • Limb Deformities, Congenital / genetics*
  • Limb Deformities, Congenital / physiopathology
  • Male
  • Membrane Proteins / genetics*
  • Mutation
  • Saudi Arabia
  • Scalp Dermatoses / congenital*
  • Scalp Dermatoses / genetics
  • Scalp Dermatoses / physiopathology
  • T-Box Domain Proteins / genetics*
  • rac1 GTP-Binding Protein / genetics*

Substances

  • LMBR1 protein, human
  • Membrane Proteins
  • RAC1 protein, human
  • T-Box Domain Proteins
  • T-box transcription factor 5
  • TBX3 protein, human
  • rac1 GTP-Binding Protein

Supplementary concepts

  • Adams Oliver syndrome