Evidence that paternal expression of the epsilon-sarcoglycan gene accounts for reduced penetrance in myoclonus-dystonia

Am J Hum Genet. 2002 Dec;71(6):1303-11. doi: 10.1086/344531. Epub 2002 Nov 20.

Abstract

Myoclonus-dystonia (M-D) is a movement disorder characterized by rapid muscle contractions and sustained twisting and repetitive movements and has recently been associated with mutations in the epsilon-sarcoglycan gene (SGCE). The mode of inheritance is autosomal dominant with reduced penetrance upon maternal transmission, suggesting a putative maternal imprinting mechanism. We present an apparently sporadic M-D case and two patients from an M-D family with seemingly autosomal recessive inheritance. In both families, we detected an SGCE mutation that was inherited from the patients' clinically unaffected fathers in an autosomal dominant fashion. Whereas, in the first family, RNA expression studies revealed expression of only the mutated allele in affected individuals and expression of the normal allele exclusively in unaffected mutation carriers, the affected individual of the second family expressed both alleles. In addition, we identified differentially methylated regions in the promoter region of the SGCE gene as a characteristic feature of imprinted genes. Using a rare polymorphism in the promoter region in a family unaffected with M-D as a marker, we demonstrated methylation of the maternal allele, in keeping with maternal imprinting of the SGCE gene. Loss of imprinting in the patient with M-D who had biallelic expression of the SGCE gene was associated with partial loss of methylation at several CpG dinucleotides.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Child
  • Child, Preschool
  • CpG Islands / genetics
  • Cytoskeletal Proteins / genetics*
  • DNA Methylation
  • DNA Mutational Analysis
  • Dystonia / complications*
  • Dystonia / genetics*
  • Fathers
  • Female
  • Genes, Dominant / genetics
  • Genomic Imprinting
  • Haplotypes / genetics
  • Humans
  • Male
  • Membrane Glycoproteins / genetics*
  • Molecular Sequence Data
  • Mutation / genetics*
  • Myoclonus / complications*
  • Myoclonus / genetics*
  • Pedigree
  • Penetrance
  • Polymorphism, Genetic / genetics
  • Promoter Regions, Genetic / genetics
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Sarcoglycans

Substances

  • Cytoskeletal Proteins
  • Membrane Glycoproteins
  • RNA, Messenger
  • Sarcoglycans

Associated data

  • OMIM/159900
  • OMIM/604149
  • RefSeq/NM_003919