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Epigenetic Drugs: Taking it to the Clinic- An Up-and-Coming Mode of Targeting Disease
Session Chair(s)
Philip (P.J.) Brooks, PhD
Deputy Director, Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences (NCATS), NIH, United States
When we thinking about the genome, we think of the DNA double helix, and the 4 canonical nitrogenous bases. The epigenome encompasses chemical modifications of DNA bases, most commonly on cytosine, as well as chemical modifications of the histone proteins that compress nuclear DNA into chromatin. Understanding the functional significance of these modifications continues to be, an active area of research investigation, but broadly speaking, chemical modifications of the epigenome influence gene expression. As such, the ability to modify the epigenome is becoming an active area of therapeutic development. In this session will focus on a basic understanding of the epigenome, as well as two very different therapeutic strategies: one focusing on small molecule drugs, and the other using epigenome editing.
Learning Objective : Describe the basic components of the epigenome, and functional consequences; Illustrate an example of the development of a drug to modulate the epigenome; Discuss strategies to adapt genome editing technology to modify the epigenome.
Speaker(s)
Epigenetic Drugs’ Challenging and Exciting Road to the Clinic
Sivakumar Gowrisankar, PhD
Parexel International, United States
Director, Genomic Medicine
Epigenome Editing for Gene Therapy and Functional Genomics
Charles A. Gersbach, PhD
Duke University, United States
Professor of Biomedical Engineering
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