COL6A1 genomic deletions in Bethlem myopathy and Ullrich muscular dystrophy

Ann Neurol. 2006 Jan;59(1):190-5. doi: 10.1002/ana.20705.

Abstract

We have identified highly similar heterozygous COL6A1 genomic deletions, spanning from intron 8 to exon 13 or intron 13, in two patients with Ullrich congenital muscular dystrophy and the milder Bethlem myopathy. The 5' breakpoints of both deletions are located within a minisatellite in intron 8. The mutations cause in-frame deletions of 66 and 84 amino acids in the amino terminus of the triple-helical domain, leading to intracellular accumulation of mutant polypeptides and reduced extracellular collagen VI microfibrils. Our studies identify a deletion-prone region in COL6A1 and suggest that similar mutations can lead to congenital muscle disorders of different clinical severity.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • Cells, Cultured
  • Child
  • Child, Preschool
  • Collagen Type VI / genetics*
  • DNA Mutational Analysis
  • Exons
  • Female
  • Fibroblasts / cytology
  • Fibroblasts / metabolism
  • Gene Deletion*
  • Humans
  • Infant
  • Introns
  • Male
  • Molecular Sequence Data
  • Muscular Diseases / genetics*
  • Muscular Dystrophies / genetics*
  • Mutation*

Substances

  • Collagen Type VI