Mutation of gene in spinal muscular atrophy respiratory distress type I

Pediatr Neurol. 2006 Jun;34(6):474-7. doi: 10.1016/j.pediatrneurol.2005.10.022.

Abstract

Spinal muscular atrophy with respiratory distress type I (SMARD1, MIM #604 320) is an uncommon variant of infantile spinal muscular atrophy type I. Distinguishing features include diaphragmatic palsy, early-onset distal limb wasting, and contracture. This report describes a Chinese male with typical features of spinal muscular atrophy with respiratory distress type I. Direct sequencing of the causative gene, the immunoglobulin mu-binding protein 2 (IGHMBP2) gene, revealed the presence of a novel frameshift mutation caused by deletion of G in exon 13 and a single base pair substitution of G to A in exon 12 resulting in substitution of isoleucine for valine.

Publication types

  • Case Reports

MeSH terms

  • DNA-Binding Proteins / genetics*
  • Humans
  • Infant
  • Male
  • Mutation / genetics*
  • Respiratory Insufficiency / genetics*
  • Spinal Muscular Atrophies of Childhood / complications
  • Spinal Muscular Atrophies of Childhood / diagnosis*
  • Spinal Muscular Atrophies of Childhood / genetics*
  • Transcription Factors / genetics*

Substances

  • DNA-Binding Proteins
  • IGHMBP2 protein, human
  • Transcription Factors