A novel de novo STXBP1 mutation is associated with mitochondrial complex I deficiency and late-onset juvenile-onset parkinsonism

Neurogenetics. 2015 Jan;16(1):65-7. doi: 10.1007/s10048-014-0431-z. Epub 2014 Nov 25.

Abstract

Mutations in STXBP1 have recently been identified as a cause of infantile epileptic encephalopathy. The underlying mechanism of the disorder remains unclear and, recently, several case reports have described broad and progressive neurological phenotypes in addition to early-onset epilepsy. Herein, we describe a patient with early-onset epilepsy who subsequently developed a progressive neurological phenotype including parkinsonism in her early teens. A de novo mutation in STXBP1 (c.416C>T, p.(Pro139Leu)) was detected with exome sequencing together with profound impairment of complex I of the mitochondrial respiratory chain on muscle biopsy. These findings implicate a secondary impairment of mitochondrial function in the progressive nature of the disease phenotype.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Brain / physiopathology
  • Child
  • Disease Progression
  • Electroencephalography
  • Electron Transport Complex I / deficiency*
  • Electron Transport Complex I / genetics
  • Epilepsy / complications
  • Epilepsy / genetics*
  • Exome
  • Female
  • Humans
  • Mitochondrial Diseases / complications
  • Mitochondrial Diseases / genetics*
  • Munc18 Proteins / genetics*
  • Mutation, Missense*
  • Parkinsonian Disorders / complications
  • Parkinsonian Disorders / genetics*
  • Phenotype

Substances

  • Munc18 Proteins
  • STXBP1 protein, human
  • Electron Transport Complex I

Supplementary concepts

  • Mitochondrial complex I deficiency