Type B mandibuloacral dysplasia with congenital myopathy due to homozygous ZMPSTE24 missense mutation

Eur J Hum Genet. 2011 Jun;19(6):647-54. doi: 10.1038/ejhg.2010.256. Epub 2011 Jan 26.

Abstract

Mutation in ZMPSTE24 gene, encoding a major metalloprotease, leads to defective prelamin A processing and causes type B mandibuloacral dysplasia, as well as the lethal neonatal restrictive dermopathy syndrome. Phenotype severity is correlated with the residual enzyme activity of ZMPSTE24 and accumulation of prelamin A. We had previously demonstrated that a complete loss of function in ZMPSTE24 was lethal in the neonatal period, whereas compound heterozygous mutations including one PTC and one missense mutation were associated with type B mandibuloacral dysplasia. In this study, we report a 30-year longitudinal clinical survey of a patient harboring a novel severe and complex phenotype, combining an early-onset progeroid syndrome and a congenital myopathy with fiber-type disproportion. A unique homozygous missense ZMPSTE24 mutation (c.281T>C, p.Leu94Pro) was identified and predicted to produce two possible ZMPSTE24 conformations, leading to a partial loss of function. Western blot analysis revealed a major reduction of ZMPSTE24, together with the presence of unprocessed prelamin A and decreased levels of lamin A, in the patient's primary skin fibroblasts. These cells exhibited significant reductions in lifespan associated with major abnormalities of the nuclear shape and structure. This is the first report of MAD presenting with confirmed myopathic abnormalities associated with ZMPSTE24 defects, extending the clinical spectrum of ZMPSTE24 gene mutations. Moreover, our results suggest that defective prelamin A processing affects muscle regeneration and development, thus providing new insights into the disease mechanism of prelamin A-defective associated syndromes in general.

Publication types

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

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Abnormalities, Multiple / physiopathology
  • Acro-Osteolysis / complications
  • Acro-Osteolysis / genetics*
  • Acro-Osteolysis / physiopathology
  • Adult
  • Amino Acid Sequence
  • Cell Culture Techniques
  • Female
  • Fibroblasts
  • Heterozygote
  • Homozygote
  • Humans
  • Lamin Type A / genetics*
  • Lamin Type A / metabolism
  • Lipodystrophy / complications
  • Lipodystrophy / genetics*
  • Lipodystrophy / physiopathology
  • Mandible / abnormalities
  • Mandible / physiopathology
  • Membrane Proteins / genetics*
  • Membrane Proteins / metabolism
  • Metalloendopeptidases / genetics*
  • Metalloendopeptidases / metabolism
  • Molecular Sequence Data
  • Muscular Dystrophies / complications
  • Muscular Dystrophies / genetics*
  • Muscular Dystrophies / physiopathology
  • Mutation
  • Mutation, Missense
  • Myopathies, Structural, Congenital / complications
  • Myopathies, Structural, Congenital / genetics*
  • Myopathies, Structural, Congenital / physiopathology
  • Nuclear Proteins / genetics*
  • Nuclear Proteins / metabolism
  • Phenotype
  • Progeria / complications
  • Progeria / genetics*
  • Progeria / physiopathology
  • Protein Precursors / genetics*
  • Protein Precursors / metabolism

Substances

  • Lamin Type A
  • Membrane Proteins
  • Nuclear Proteins
  • Protein Precursors
  • prelamin A
  • Metalloendopeptidases
  • ZMPSTE24 protein, human

Supplementary concepts

  • Mandibuloacral dysplasia with type A lipodystrophy