Pharmacological and Therapeutic Targeting of Epigenetic Regulators

Raul Urrutia, Gwen A. Lomberk

Research output: Chapter in Book/Report/Conference proceedingChapter


Genetics has been transformational for understanding causes and mechanisms of human diseases, developing diagnostics approaches, and providing indirect therapeutic interventions that target molecule functions upstream and downstream of mutated genes. Significant efforts have been given to correct genetic alterations or deliver genes in the clinical setting by gene therapy approaches. However, in spite of early promises and current significant advances, gene therapy has not yet brought useful protocols to the bedside for diseases that are multigenic in nature, such as cancer. Epigenetics is emerging as a revolutionary science in biology, which studies mechanisms that regulate entire gene networks to give rise to and maintain phenotypes independent of the coding capacity of DNA. In fact, epigenetic alterations appear to be more common than genetics as a cause of chronic diseases by silencing or activating single genes or entire gene networks. More importantly, epigenetic alterations can, by definition, be inherited, thereby propagating disease-prone phenotypes. Fortunately, in the last decade, extensive investigations have resulted in the development of small drugs that modify the epigenome in a reversible manner. This chapter will summarize the most conspicuous advances in this field and discuss how epigenetic pharmacology is becoming a solid foundation for mechanistic experiments and new therapies.

Original languageEnglish (US)
Title of host publicationChromatin Signaling and Diseases
PublisherElsevier Inc.
Number of pages15
ISBN (Electronic)9780128026090
ISBN (Print)9780128023891
StatePublished - Aug 24 2016


  • DNA methylation
  • Epigenetics
  • Histone-modifying enzymes
  • Non-coding RNA
  • Pharmacology
  • Therapeutics

ASJC Scopus subject areas

  • General Biochemistry, Genetics and Molecular Biology


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