In junk we trust: repetitive DNA, epigenetics and facioscapulohumeral muscular dystrophy

Epigenomics. 2010 Apr;2(2):271-87. doi: 10.2217/epi.10.8.

Abstract

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant myopathy with a peculiar etiology. Unlike most genetic disorders, FSHD is not caused by mutations in a protein-coding gene. Instead, it is associated with contraction of the D4Z4 macrosatellite repeat array located at 4q35. Interestingly, D4Z4 deletion is not sufficient per se to cause FSHD. Moreover, the disease severity, its rate of progression and the distribution of muscle weakness display great variability even among close family relatives. Hence, additional genetic and epigenetic events appear to be required for FSHD pathogenesis. Indeed, recent findings suggest that virtually all levels of epigenetic regulation, from DNA methylation to higher order chromosomal architecture, exhibit alterations in the disease locus causing deregulation of 4q35 gene expression, ultimately leading to FSHD.

Publication types

  • Review

MeSH terms

  • Chromatin / genetics
  • Chromosomes, Human, Pair 4 / genetics*
  • Epigenesis, Genetic / genetics
  • Epigenesis, Genetic / physiology*
  • Gene Expression Regulation / genetics
  • Gene Expression Regulation / physiology*
  • Homeodomain Proteins / genetics
  • Humans
  • Microfilament Proteins
  • Microsatellite Repeats / genetics*
  • Muscular Dystrophy, Facioscapulohumeral / genetics*
  • Muscular Dystrophy, Facioscapulohumeral / physiopathology
  • Nuclear Proteins / genetics
  • RNA-Binding Proteins
  • Repetitive Sequences, Nucleic Acid / genetics*

Substances

  • Chromatin
  • DUX4L1 protein, human
  • FRG1 protein, human
  • Homeodomain Proteins
  • Microfilament Proteins
  • Nuclear Proteins
  • RNA-Binding Proteins