Base Editing and Prime Editing: Precise Genome Modification Without DNA Breaks

Explore the cutting-edge developments in precision gene editing technologies presented by David Liu in this seminar from the Broad-MIT Seminar Series in Chemical Biology. Delve into the world of base editing and prime editing, revolutionary techniques that allow for precise DNA modifications without causing double-strand breaks. Learn about the advantages of these methods in minimizing undesired consequences such as translocations and chromothripsis. Discover how base editors have been engineered to work independently of CRISPR, enabling the first intentional changes to mitochondrial DNA in living cells. Examine the therapeutic applications of these technologies, including their use in animal models to treat genetic diseases like sickle-cell disease and progeria. Gain insights into the development of single-AAV base editing systems and engineered virus-like particles (eVLPs) for improved in vivo delivery of gene editing proteins. Understand the principles behind prime editors and their ability to write new genetic information into specific DNA sites without requiring donor DNA templates. Investigate the cellular determinants of prime editing outcomes and the recent advancements in improving editing efficiencies. Explore the potential of combining prime editing with recombinases for programmable gene-sized integration and inversion in human cells. Grasp the broad implications of these technologies for life sciences and therapeutics, including their current use in clinical trials for various genetic disorders.