Duplication of dystrophin gene and dissimilar clinical phenotype in the same family

Neuromuscul Disord. 1995 Nov;5(6):475-81. doi: 10.1016/0960-8966(95)00008-b.

Abstract

We report here three related patients with a duplication of exons 19-41 of the dystrophin gene, having dissimilar clinical phenotype and dystrophin immunohistochemistry. Two brothers aged six and three years had myalgia, proximal muscular weakness and hypertrophic calves, with 10- 20-fold increase of serum creatine kinase. Muscle biopsy showed dystrophic changes and reduced, patchy binding of dystrophin. The clinical and laboratory findings were consistent with a diagnosis of Becker muscular dystrophy with early onset. Their 14-year-old cousin had only mild hyperCKemia. His muscle biopsy was normal with only mild reduction of dystrophin immunostaining. At follow-up, he is still without symptoms and signs at age 19. All three patients had the same gene duplication and an increased dystrophin size of 507 kDa. Expression of the dystrophin-associated glycoproteins adhalin, alpha-dystroglycan, and beta-dystroglycan were normal in the three patients. An intrafamilial variability in patients carrying a partial duplication of the dystrophin gene may be related to a quantitative difference in mRNA.

Publication types

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

MeSH terms

  • Blotting, Southern
  • Blotting, Western
  • Child
  • Child, Preschool
  • DNA / biosynthesis
  • Dystrophin / analysis
  • Dystrophin / genetics*
  • Dystrophin / metabolism
  • Exons
  • Fluorescent Antibody Technique
  • Humans
  • Immunohistochemistry
  • Male
  • Multigene Family / genetics*
  • Muscle, Skeletal / pathology
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / metabolism
  • Muscular Dystrophies / pathology
  • Phenotype

Substances

  • Dystrophin
  • DNA

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