Editing the Epigenome to Tackle Brain Disorders

Trends Neurosci. 2019 Dec;42(12):861-870. doi: 10.1016/j.tins.2019.10.003. Epub 2019 Nov 7.


Genetic studies of epigenetic modifiers such as DNA methyltransferases and histone acetyltransferases have revealed a critical role for epigenetic regulation during brain development and function. Alteration of epigenetic modifications have been documented in a variety of brain disorders, including neurodevelopmental, psychiatric, and neurodegenerative diseases. Development of epigenome editing tools enables a functional dissection of the link between altered epigenetic changes and disease outcomes. Here, we review the development of epigenome editing tools, summarize proof of concept applications focusing on brain disease-associated genes, and discuss the promising application and challenges of epigenome editing to tackle brain disorders.

Keywords: CRISPR; brain disorders; dCas9; epigenome editing.

Publication types

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

MeSH terms

  • Animals
  • Brain Diseases / genetics*
  • CRISPR-Cas Systems*
  • DNA Methylation
  • Epigenesis, Genetic*
  • Epigenomics / methods*
  • Gene Editing / methods*
  • Humans