Angelman syndrome: Drugs to awaken a paternal gene

Nature. 2011 Dec 21;481(7380):150-2. doi: 10.1038/nature10784.

Abstract

Mutations in the maternal copy of the UBE3A gene cause a neurodevelopmental disorder known as Angelman syndrome. Drugs that activate the normally silenced paternal copy of this gene may be of therapeutic value.

Publication types

  • News
  • Comment

MeSH terms

  • Alleles*
  • Animals
  • Female
  • Gene Silencing / drug effects*
  • Male
  • Neurons / drug effects*
  • Neurons / metabolism*
  • Topoisomerase Inhibitors / pharmacology*
  • Ubiquitin-Protein Ligases / genetics*

Substances

  • Topoisomerase Inhibitors
  • Ube3a protein, mouse
  • Ubiquitin-Protein Ligases