Epigenetic editing: from concept to clinic
Summary
Dysregulated gene expression is associated with nearly all human disease and can be mediated by epigenetic modifications. Epigenetic editing aims to reprogramme gene expression by rewriting epigenetic signatures, without editing of the genome. Coined just over a decade ago, epigenetic editing initially faced major concerns regarding efficacy and specificity. Moreover, straightforward guidelines on how to induce sustained expression modulation for any given gene are still largely lacking. Y
Content
# Epigenetic editing: from concept to clinic
*Published: 2026 Mar*
Dysregulated gene expression is associated with nearly all human disease and can
be mediated by epigenetic modifications. Epigenetic editing aims to reprogramme
gene expression by rewriting epigenetic signatures, without editing of the
genome. Coined just over a decade ago, epigenetic editing initially faced major
concerns regarding efficacy and specificity. Moreover, straightforward
guidelines on how to induce sustained expression modulation for any given gene
are still largely lacking. Yet, successes have been demonstrated in animal
models of various diseases, and the first clinical trials of epigenetic editing
have been initiated. Increased understanding of the mechanisms of epigenetic
reprogramming is overcoming obstacles that initially hampered widespread
application of epigenetic editing. Future advances in target specificity,
reprogramming maintenance and delivery methods will enable epigenetic editing to
become a powerful new therapeutic approach.
DOI: 10.1038/s41573-025-01323-0