Unusual Charcot-Marie-Tooth phenotype due to a mutation within the intracellular domain of myelin protein zero

Muscle Nerve. 2010 Apr;41(4):550-4. doi: 10.1002/mus.21523.

Abstract

Myelin protein zero (MPZ/P0) constitutes a major component of compact peripheral myelin. We report a family with a missense mutation, c.700G>T p.Asp234Tyr (deviant nomenclature: c.670G>T, p.Asp224Tyr), within the intracellular domain of myelin protein zero, who has distal sensorimotor symptoms, cramps, restless legs syndrome, neuropathic pain, and carpal tunnel syndrome. The index patient responded to intravenous immunoglobulin and immunosuppression, so there may be a possible secondary autoimmune process, probably triggered by altered antigen presentation due to mutated MPZ protein.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Charcot-Marie-Tooth Disease / diagnosis
  • Charcot-Marie-Tooth Disease / genetics*
  • Female
  • Humans
  • Intracellular Fluid* / chemistry
  • Intracellular Fluid* / metabolism
  • Middle Aged
  • Mutation, Missense / genetics*
  • Myelin P0 Protein / genetics*
  • Pedigree
  • Phenotype*
  • Protein Structure, Tertiary / genetics

Substances

  • Myelin P0 Protein