Substitution of a conserved cysteine-996 in a cysteine-rich motif of the laminin alpha2-chain in congenital muscular dystrophy with partial deficiency of the protein

Am J Hum Genet. 1996 Jun;58(6):1177-84.

Abstract

Congenital muscular dystrophies (CMDs) are autosomal recessive muscle disorders of early onset. Approximately half of CMD patients present laminin alpha2-chain (merosin) deficiency in muscle biopsies, and the disease locus has been mapped to the region of the LAMA2 gene (6q22-23) in several families. Recently, two nonsense mutations in the laminin alpha2-chain gene were identified in CMD patients exhibiting complete deficiency of the laminin alpha2-chain in muscle biopsies. However, a subset of CMD patients with linkage to LAMA2 show only partial absence of the laminin alpha2-chain around muscle fibers, by immunocytochemical analysis. In the present study we have identified a homozygous missense mutation in the alpha2-chain gene of a consanguineous Turkish family with partial laminin alpha2-chain deficiency. The T-->C transition at position 3035 in the cDNA sequence results in a Cys996-->Arg substitution. The mutation that affects one of the conserved cysteine-rich repeats in the short arm of the laminin alpha2-chain should result in normal synthesis of the chain and in formation and secretion of a heterotrimeric laminin molecule. Muscular dysfunction is possibly caused either by abnormal disulfide cross-links and folding of the laminin repeat, leading to the disturbance of an as yet unknown binding function of the laminin alpha2-chain and to shorter half-life of the muscle-specific laminin-2 and laminin-4 isoforms, or by increased proteolytic sensitivity, leading to truncation of the short arm.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Base Sequence
  • Brain / pathology
  • Child, Preschool
  • Chromosome Mapping
  • Chromosomes, Human, Pair 6*
  • Consanguinity
  • Consensus Sequence
  • Conserved Sequence
  • Cysteine*
  • DNA / chemistry
  • DNA Primers
  • Drosophila
  • Female
  • Genetic Carrier Screening
  • Genetic Linkage
  • Homozygote
  • Humans
  • Laminin / deficiency*
  • Laminin / genetics*
  • Magnetic Resonance Imaging
  • Male
  • Mice
  • Molecular Sequence Data
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • Muscular Dystrophies / congenital
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / pathology
  • Pedigree
  • Point Mutation*
  • Polymorphism, Single-Stranded Conformational
  • Sequence Homology, Amino Acid

Substances

  • DNA Primers
  • Laminin
  • DNA
  • Cysteine