Use of epigenetic drugs in disease: an overview

Sarah Heerboth1, Karolina Lapinska1, Nicole Snyder1

  • 1Cancer Center, Department of Medicine, Boston University School of Medicine, Boston, MA, USA.

Genetics & Epigenetics
|December 17, 2014
PubMed

Insights

Epigenetic drugs target DNA methylation and histone modifications to reverse pathological gene expression changes. This review covers epigenetic events in diseases and discusses key drug classes for therapeutic intervention.

Area of Science:

  • Molecular Biology
  • Pharmacology
  • Genetics

Background:

  • Epigenetic alterations like DNA methylation and histone modifications regulate gene expression.
  • Dysregulation of these epigenetic events is linked to various diseases, including cancer, cardiovascular, neurological, and metabolic disorders.
  • Targeting epigenetic modifications offers a promising therapeutic strategy.

Purpose of the Study:

  • To review epigenetic events associated with major human diseases.
  • To discuss the development and therapeutic potential of epigenetic drugs.
  • To highlight key classes of epigenetic inhibitors.

Main Methods:

  • Literature review of epigenetic mechanisms in disease.
  • Analysis of current epigenetic drug classes and their targets.
  • Discussion of therapeutic applications in cardiovascular, neurological, metabolic disorders, and cancer.

Main Results:

  • Specific epigenetic changes are implicated in diverse pathologies.
  • Epigenetic drugs, including DNA methylation inhibitors and histone-modifying enzyme inhibitors, show therapeutic promise.
  • Bromodomain inhibitors, histone acetyl transferase inhibitors, histone deacetylase inhibitors, and protein methyltransferase inhibitors represent major classes.

Conclusions:

  • Understanding disease-specific epigenetic alterations is crucial for developing targeted therapies.
  • Epigenetic drugs offer a novel approach to reverse pathological gene expression and treat diseases.
  • Further research into epigenetic drug development holds significant clinical potential.

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