Seminar on Neurology_Development of Novel Genome Editing Tools by Combining Structural and Synthetic Biology

Event Details

The development of novel genome editing tools by combining structural biology and synthetic biology.
Speaker] Dr. Osamu Nureki, Graduate School of Science, The University of Tokyo Professor

Gene therapies based on genome editing have been approved one after another around the world. Streptococcus pyogenes-derived Cas9 (SpCas9) has been widely used because of its high genome editing activity, but it has a fatal drawback. Therefore, we have developed SpCas9-NG and SaCas9-NNG from Staphylococcus aureus, which recognize a single guanine letter, by theoretical design based on the three-dimensional structure. Furthermore, we have successfully created an activity-enhancing mutant (enAsCas12f) by combining Deep Mutational Scanning (artificial evolution) and structure-based design. Furthermore, enAsCas12f packaged in a single AAV vector showed efficient knock-in/knock-out activity and transcriptional activation in mice. Thus, structure-based mutagenesis has paved the way for the development of the ultimate genome editing tool that can correct any genomic mutation.

References) Science 2018, 361, 1259-1262, Cell 2023, 186, 4920-4935; Nature in press.

Courses for which credit is granted
● Graduate School of Medical Sciences (doctoral course) Division of Medicine
Course Title "Up-to-date Seminars"

● School of Medicine
Course Title "Latest Medical Research

Contact

Kanazawa University Faculty of Medicine Division of Neurology Hiroshi Kawasaki, kawasaki-labo@umin.ac.jp, Faculty of Health Sciences Pathophysiology Yuhei Araiso, araiso@staff.kanazawa-u.ac.jp