The molecular basis of succinic semialdehyde dehydrogenase deficiency in one family

Mol Genet Metab. 2004 Apr;81(4):347-51. doi: 10.1016/j.ymgme.2004.01.012.

Abstract

Succinic semialdehyde dehydrogenase (SSADH) deficiency has predominantly neurological consequences, affecting psychomotor, speech and language development. Recently, two clinical reviews summarized the features of this disease and their relative frequency [Neurology 60 (2003) 1413; Ann. Neurol. 54 (2003) S73]. The molecular genetics of SSADH deficiency is still being explored. We describe the molecular basis of this defect in a Tunisian female child presenting with a mild phenotype. A small scale deletion in exon 10 of the gene led to a frameshift that predicts premature termination of the resulting putative protein. The parents were shown to be heterozygotes for this deletion, supporting its causative role.

Publication types

  • Case Reports

MeSH terms

  • Aldehyde Oxidoreductases / deficiency*
  • Aldehyde Oxidoreductases / genetics
  • Base Sequence*
  • Child
  • DNA Mutational Analysis
  • Female
  • Humans
  • Metabolism, Inborn Errors / genetics*
  • Molecular Sequence Data
  • Mutation
  • Phenotype
  • Sequence Deletion*
  • Succinate-Semialdehyde Dehydrogenase

Substances

  • Aldehyde Oxidoreductases
  • ALDH5A1 protein, human
  • Succinate-Semialdehyde Dehydrogenase