Mutations of PTPN23 in developmental and epileptic encephalopathy

Hum Genet. 2017 Nov;136(11-12):1455-1461. doi: 10.1007/s00439-017-1850-3. Epub 2017 Oct 31.

Abstract

Developmental and epileptic encephalopathies (DEE) are a heterogeneous group of neurodevelopmental disorders with poor prognosis. Recent discoveries have greatly expanded the repertoire of genes that are mutated in epileptic encephalopathies and DEE, often in a de novo fashion, but in many patients, the disease remains molecularly uncharacterized. Here, we describe a new form of DEE in patients with likely deleterious biallelic variants in PTPN23. The phenotype is characterized by early onset drug-resistant epilepsy, severe and global developmental delay, microcephaly, and sometimes premature death. PTPN23 encodes a tyrosine phosphatase with strong brain expression, and its knockout in mouse is embryonically lethal. Structural modeling supports a deleterious effect of the identified alleles. Our data suggest that PTPN23 mutations cause a rare severe form of autosomal-recessive DEE in humans, a finding that requires confirmation.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Developmental Disabilities / genetics*
  • Developmental Disabilities / pathology
  • Female
  • Humans
  • Infant, Newborn
  • Male
  • Mutation*
  • Phenotype
  • Protein Conformation
  • Protein Tyrosine Phosphatases, Non-Receptor / chemistry
  • Protein Tyrosine Phosphatases, Non-Receptor / genetics*
  • Spasms, Infantile / genetics*
  • Spasms, Infantile / pathology

Substances

  • PTPN23 protein, human
  • Protein Tyrosine Phosphatases, Non-Receptor

Supplementary concepts

  • Infantile Epileptic-Dyskinetic Encephalopathy