Research Seminar: Precision Genome Editing In Human iPS Cells For Disease Modeling And Improved Cell Therapies – Dr. Knut Woltjen

Meeting ID: 693 4383 4453
Passcode: 608808

Location:
Lecture Theatre B1001, Gordon B. Shrum Building (SHRM)

Human induced pluripotent stem (iPS) cells carry the genome and variants of their donor, enabling personalized in vitro models of human health and autologous cell therapies. Combined with CRISPR-Cas9 genome editing, the effects of genetic variations on human characteristics including disease predisposition and severity can be functionally tested in iPS cell-derived tissues, and genetic mutations may be corrected prior to therapeutic application. Moreover, combining knowledge of natural variant functions with precision gene editing techniques such as Prime Editing, MMEJ, DNA transposons and recombinases enables the generation of designer cells. In this seminar, I will provide examples of precision genome editing in human iPS cells for diverse applications including improved cell differentiation and production in vitro, immune evasion for broad application of iPS cell stocks, and functional elucidation of “dark”, repetitive regions of the genome such as Variable Number Tandem Repeats (VNTRs) which are polymorphic in human populations. I will also introduce the i²PARADIGM network, which brings together international expertise in next-generation technologies including genome sequencing, personal iPS cells, gene and tissue engineering, and semiconductor technology to foster the next generation of researchers in personalized or n-of-1 therapeutic development.

Dr. Knut Woltjen’s Biography:

Dr. Woltjen is an Associate Professor in the Center for iPS Cell Research and Application (CiRA) and alumnus of the Hakubi Center for Advanced Research of Kyoto University, Japan. He received his B.Sc. (Honours) in Molecular Genetics and his Ph.D. thesis focused on the development of gene editing technologies for mouse and human embryonic stem (ES) cells. As a post-doctoral fellow, Dr. Woltjen employed the piggyBac transposon to create the first footprint-free mouse and human induced pluripotent stem cell (iPS) cells. In CiRA, Dr. Woltjen and his colleagues have continued to use piggyBac to direct the differentiation of iPS cells into cells such as muscle and neurons, as well as achieve precision gene editing. Dr. Woltjen’s lab demonstrated the utility of microhomology-mediated end joining (MMEJ) to create human pathogenic deletions and precise edits. Dr. Woltjen’s ongoing research focuses on developing and applying methods for precision genome and epigenome editing to improve cell therapies as well as understand and control the genetics of human health, disease, and evolution.