Abstract:

In this presentation, I will provide an overview of gene-editing technologies, focusing on the fundamentals of CRISPR-Cas9, base editors, and prime editors. I will explore the underlying mechanisms of each tool, highlighting their unique capabilities and advancements. CRISPR-Cas9, widely recognized for its ability to introduce precise double-strand breaks, revolutionized genetic manipulation. Building on this, base editors enable single-nucleotide changes without double-strand breaks, offering a more refined approach for correcting point mutations. The latest innovation, prime editing, combines CRISPR-Cas9 and reverse transcriptase, allowing precise editing of targeted DNA sequences with minimal off-target effects. I will also discuss the therapeutic applications of these tools, including their potential in developing treatments for genetic disorders. Particular attention will be given to prime editing, which shows promise in addressing previously intractable mutations. Furthermore, I will delve into the use of prime editors for functional evaluation of variants of uncertain significance (VUS). This method provides crucial insights into the pathogenicity of VUS, contributing to more accurate genetic diagnoses and personalized medicine. By bridging the gap between cutting-edge gene editing and clinical application, this talk aims to shed light on the future of genomic medicine.

Speaker’s bio:

Kim is a professor in the Department of Pharmacology, Yonsei University college of Medicine, Seoul, South Korea. He received his M.D. in 2001 and Ph.D. in 2006 from Yonsei University, Seoul. During his Ph.D. programme, he studied tissue engineering using mesenchymal stem cells and biomaterials. After postdoctoral training at Emory University, Atlanta, Georgia, USA, in the field of stem cell biology, he became an independent researcher in 2010, when he changed his research field to genome editing.

His group developed a high-throughput method for profiling genome editors including Cpf1 (Cas12a), various Cas9s, base editors, and prime editors. Using the resulting large-scale datasets of genome editor activities, his group developed deep learning models to predict these various genome editor activities with high accuracy. His group has also generated a CRISPR clock that enables recording elapsed time into integrated DNA sequences of live mammalian cells. Ongoing projects in his lab include the functional evaluation of variants of uncertain significance and the development of potential therapeutic applications of genome editors for various diseases. He is a co-founder and chief scientific advisor of a biotechnology company named LiquidCRISPR.