The Open BME Seminar Series is coordinated by seven of the leading BME departments in the country.
This virtual seminar series is being coordinated by seven of the leading BME departments in the country. The goal is to expose our community to cutting edge ideas in the field of biomedical engineering by attracting a group of high-quality speakers that would be difficult for any single department to host on its own.
Participating BME departments are the Univerity of Virginia, Case Western Reserve University, Northwestern University, Rice University, University of Michigan, University of Pittsburgh, and Washington University in St. Louis.
February 18th, 4-5pm EST
GENE TARGETING OF STEM CELLS TO CREATE EFFECTIVE AND SAFE MEDICINES
Matthew Porteus, MD, PhD, Professor, Pediatrics-Stem Cell Transplantation, Stanford School of Medicine
Hosted by Dr. Gang Bao | Rice University
Genome editing is a powerful approach to genetically engineer cells with single nucleotide precision. The precision can be used in a wide variety of ways to generate cells that could cure patients of disease. In this talk, I will discuss how we have developed a CRISPR/Cas9 based system that can not only correct single nucleotide disease causing mutations, but can also integrate gene cassettes at specific locations. We have applied this system to a variety of stem cells, including pluripotent and somatic, to leverage the biologic properties of stem cells to regenerate organ systems, including the blood and immune systems. In addition, genome editing can also be combined with the principles of synthetic biology to engineer cells with new therapeutic functions, including for both improved safety and efficacy. Finally, I will end by discussing the biologic and engineering challenges to applying targeted integration genome editing in vivo.
Matthew Porteus MD, PhD is a Professor in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine and Maternal-Child Health Research Institute at Stanford. He is the co-Director for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (such as sickle cell disease) but also of other organ systems as well. His goal is to combine his research and clinical interests to develop innovative curative therapies. And his dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NExTRAC advisory committee evaluating the emergence of new technologies.