A Coordinated Attack: Rett Syndrome Therapeutic Development

Trends Pharmacol Sci. 2019 Apr;40(4):233-236. doi: 10.1016/j.tips.2019.02.007.

Abstract

Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the Methyl CpG binding protein 2 (MeCP2) gene. This Science & Society article focuses on pharmacological strategies that attack RTT treatment from multiple angles, including drug repurposing and de novo discovery efforts, and discusses the impacts of preclinical study design and translationally relevant outcome measures.

Keywords: Rett syndrome; pharmacological intervention.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Drug Discovery / methods*
  • Drug Evaluation, Preclinical / methods
  • Drug Repositioning
  • Female
  • Humans
  • Methyl-CpG-Binding Protein 2 / genetics*
  • Mutation
  • Research Design
  • Rett Syndrome / drug therapy*
  • Rett Syndrome / genetics
  • Rett Syndrome / physiopathology

Substances

  • MECP2 protein, human
  • Methyl-CpG-Binding Protein 2