Missense mutations in exon 6 of the survival motor neuron gene in patients with spinal muscular atrophy (SMA)

Hum Mol Genet. 1997 May;6(5):821-5. doi: 10.1093/hmg/6.5.821.

Abstract

Spinal muscular atrophy (SMA) is a frequent autosomal recessive neurodegenerative disorder leading to weakness and atrophy of voluntary muscles. The survival motor neuron gene (SMN) is a strong candidate for SMA and present in two highly homologous copies (telSMN and cenSMN) within the SMA region (5q11.2-q13.3). More than 90% of SMA patients show homozygous deletions of at least exon 7 of telSMN, whereas absence of cenSMN seems to have no clinical consequences. In 23 non-deleted SMA patients, we searched for intragenic mutations of the SMN genes in exons 1-7 and the promotor region by single strand conformation analysis. We identified two different missense mutations, S2621 and T2741, in exon 6 of telSMN in three independent SMA families, providing further evidence for the telSMN gene as a SMA determining gene. Both mutations, as well as two previously described mutations (Y272C and G279V) are located within a highly conserved interval from codon 258 to codon 279 which seems to be an important functional domain of the telSMN protein. Recently, this region has been shown to contain a tyrosine/glycine-rich motif, which is also present in various RNA binding proteins, suggesting a potential role of SMN in RNA metabolism. Missense mutations might be useful for in vivo and transgenic experiments and further investigations on understanding the function of the telSMN protein.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Alternative Splicing
  • Child
  • Child, Preschool
  • Cyclic AMP Response Element-Binding Protein
  • Exons
  • Female
  • Genetic Markers
  • Haplotypes
  • Humans
  • Infant
  • Male
  • Muscular Atrophy, Spinal / genetics*
  • Mutation*
  • Nerve Tissue Proteins / genetics*
  • Pedigree
  • Polymorphism, Single-Stranded Conformational
  • RNA-Binding Proteins
  • SMN Complex Proteins
  • Sequence Analysis, DNA
  • Transcription, Genetic

Substances

  • Cyclic AMP Response Element-Binding Protein
  • Genetic Markers
  • Nerve Tissue Proteins
  • RNA-Binding Proteins
  • SMN Complex Proteins