Epigenetic Therapies in the Precision Medicine Era. Issue 8 (8th June 2020) - Leite, Michel Lopes
Abstract: The elucidation of the 3D structure of DNA revolutionized modern science and created the basis for the field of molecular biology. The advent of DNA sequencing, and further refinement with Next‐Generation Sequencing (NGS) techniques, has made possible the enormous accumulation of data, which are useful for understanding the molecular mechanisms associated to various complex and rare diseases. Thanks to these advances, today it is known that several mechanisms regulate gene expression without the occurrence of mutations in the genome, a phenomenom known as Epigenetics and Epigenomics. The main mechanisms involved are DNA methylation, histone modifications, and non‐coding RNA transcription. The knowledge of these mechanisms applied to biomedicine has enabled the emergence of several fields, especially precision medicine, which is based on the genetic and epigenetic profiles of patients applied to personalized diagnostics and treatments. In this review, the history of the scientific advances that have enabled the development of precision medicine will be discussed, with a focus in epigenetics. Moreover, several molecules that have been approved for use or have the potential in epigenetic therapies (epidrugs) will also be discussed here, those of which act on targets responsible for maintaining the correct epigenetic pattern or correcting wrong patterns in diseases. Abstract : The constant improvement of next‐generation sequencing (NGS) technologies along with clinical information from patients, has enabled a revolution in the precision medicine field. Nowadays, it is possible to use an individual's genome/epigenome sequence and stratify patients based on their epigenomic profile for treatment. These new precision and individualized approaches will enable more effective treatments for people with complex and rare genetic diseases.