Two novel variants in CNTNAP1 in two siblings presenting with congenital hypotonia and hypomyelinating neuropathy

Eur J Hum Genet. 2017 Jan;25(1):150-152. doi: 10.1038/ejhg.2016.142. Epub 2016 Oct 26.

Abstract

Homozygous frameshift variants in CNTNAP1 have recently been reported in patients with arthrogryposis and abnormal axon myelination. In two brothers with severe congenital hypotonia and foot deformities, we identified compound heterozygous variants in CNTNAP1, reporting the first causative missense variant, p.(Cys323Arg). Motor nerve conductions were markedly decreased. Nerve microscopical lesions confirmed a severe hypomyelinating process and showed loss of attachment sites of the myelin loops on the axons, which could be a characteristic of Caspr loss-of-function. We discuss the pathophysiology of the myelination process and we propose to consider this disorder as a congenital hypomyelinating neuropathy.

MeSH terms

  • Arthrogryposis / genetics*
  • Arthrogryposis / physiopathology
  • Cell Adhesion Molecules, Neuronal / genetics*
  • Foot Deformities / genetics*
  • Foot Deformities / physiopathology
  • Genetic Predisposition to Disease
  • Homozygote
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Muscle Hypotonia / genetics*
  • Muscle Hypotonia / physiopathology
  • Mutation, Missense
  • Myelin Sheath / genetics
  • Siblings

Substances

  • CNTNAP1 protein, human
  • Cell Adhesion Molecules, Neuronal