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<p>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&#xa0;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.</p>

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Epigenetic editing: from concept to clinic

  • 期刊论文

  • 发表日期：

    2025年11月24日

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  • Elizabeth A. Heller,
  • Lacramioara Bintu,
  • Marianne G. Rots

摘要

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.