Novel PNPLA2 gene mutations in Chinese Han patients causing neutral lipid storage disease with myopathy

J Hum Genet. 2012 Oct;57(10):679-81. doi: 10.1038/jhg.2012.84. Epub 2012 Jul 26.

Abstract

Neutral lipid storage disease with myopathy (NLSDM) referred to those neutral lipid storage disease (NLSD) patients with myopathy but without ichthyosis. Recently, NLSDM has been attributed to mutations in the PNPLA2 gene. Until now, 19 patients with PNPLA2 mutations have been reported. In the present study, we describe the clinical and genetic findings in three Chinese patients with NLSDM. Sequence analysis of PNPLA2 gene was performed. In our patients we identified four novel mutations in the PNPLA2 gene including two splicing mutations. The identification and study of mutations found in PNPLA2 is also particularly important to define the clinical spectrum and genotype-phenotype correlations of the PNPLA2 gene.

Publication types

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

MeSH terms

  • Adult
  • Asian People / genetics*
  • Biopsy
  • DNA Mutational Analysis
  • Female
  • Genetic Association Studies
  • Humans
  • Lipase / genetics*
  • Lipid Metabolism, Inborn Errors / genetics*
  • Lipid Metabolism, Inborn Errors / pathology
  • Male
  • Middle Aged
  • Muscles / pathology
  • Muscular Atrophy / genetics
  • Muscular Atrophy / pathology
  • Muscular Diseases / genetics*
  • Muscular Diseases / pathology
  • Mutation*
  • Population Groups / genetics

Substances

  • Lipase
  • PNPLA2 protein, human

Supplementary concepts

  • Neutral Lipid Storage Disease with Myopathy