Epigenetics in Drug Discovery: Achievements and Challenges

The epigenome comprises a range of covalent modifications of DNA and histone proteins that establish chromatin structure and function. These processes regulate gene expression without altering the DNA sequence and play an important role in regulating, e.g., brain function and associated behaviors. Epigenetic changes are acquired throughout life in response to the environment exposed to and thus present a link between one’s environment and genetic landscape. Various epigenetic processes are thought to play a role in several psychiatric and neurodegenerative disorders including Alzheimer’s disease (AD). As such, the epigenome and its disease-associated signatures present a compelling drug target for the treatment of these disorders. Although studies of mental disease-related epigenetic changes are still in their infancy and face many challenges, neuroepigenetic research has contributed significantly to the AD field over the last decade. Research on epigenome-wide changes bears great potential in nominating epigenetics-related drug targets, and inclusion of epigenetic modalities in, e.g., multiomics approaches may provide more robust findings, which could be ultimately translated into potential drug development applications.