An intriguing "silent" mutation and a founder effect in antiquitin (ALDH7A1)

Ann Neurol. 2007 Oct;62(4):414-8. doi: 10.1002/ana.21206.

Abstract

Recently, alpha-aminoadipic semialdehyde (alpha-AASA) dehydrogenase deficiency was shown to cause pyridoxine-dependent epilepsy in a considerable number of patients. alpha-AASA dehydrogenase deficiency is an autosomal recessive disorder characterized by a neonatal-onset epileptic encephalopathy in which seizures are resistant to antiepileptic drugs but respond immediately to the administration of pyridoxine (OMIM 266100). Increased plasma and urinary levels of alpha-AASA are associated with pathogenic mutations in the alpha-AASA dehydrogenase (ALDH7A1/antiquitin) gene. Here, we report an intriguing "silent" mutation in ALDH7A1, a novel missense mutation and a founder mutation in a Dutch cohort (10 patients) with alpha-AASA dehydrogenase deficiency.

Publication types

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

MeSH terms

  • Adult
  • Aldehyde Dehydrogenase / genetics*
  • Epilepsy / genetics*
  • Female
  • Founder Effect*
  • Genetic Predisposition to Disease / genetics
  • Heterozygote
  • Humans
  • Male
  • Mutation

Substances

  • ALDH7A1 protein, human
  • Aldehyde Dehydrogenase