SC26: Gene Editing and Base Editing for Cell Therapies

TUESDAY, MARCH 3 | 6:30 - 9:30 PM (DINNER PROVIDED)

ABOUT THIS COURSE:

Cell-based therapies are yielding promising clinical benefits for treating many diseases, especially cancer and innovations in gene editing, and in particular the use of CRISPR and base editing has been crucial for engineered cell therapy. This course will highlight some of the new methods and strategies for efficient gene and base editing, targeted in vivo and ex vivo delivery and cell production for therapeutic use in various cells such as induced pluripotent stem cells, T cells, natural killer (NK) cells and hematopoietic cells. Clinical translation and safety concerns surrounding gene-edited cell therapies will also be discussed. The instructors will give short talks followed by open discussion with attendees, where they can elaborate on their experiences and expertise.

COURSE AGENDA:

6:00 pm Dinner Buffet

6:30 Course Introduction

6:40 Gene Editing and Blastocyst Complementation to Grow Human Organs in Pigs

Clifford Steer, MD, Professor of Medicine and, Genetics, Cell Biology, and Development, University of Minnesota Medical School

• Describe the role of gene editing and blastocyst complementation in generating chimeric livers in pigs
• Discuss the potential challenges in bringing such a technology to commercialization and the bedside
• Predict the future for organ transplantation based on current and developing technologies

7:20 Use of Base editing for Modification of Primary Lymphohematopoietic Cells

Beau Webber, PhD, Assistant Professor, Department of Pediatrics, Division of Hematology and Oncology, University of Minnesota

8:00 Dessert Break

8:15 Towards in vivo Delivery: Emerging Technologies for Therapeutic Genome Editing

Ross Wilson, PhD, Project Scientist & Principal Investigator, Innovative Genomics Institute, California Institute for Quantitative Biosciences, University of California, Berkeley

• Readily administered genome editing therapeutics could transform the clinical landscape, and several approaches hold substantial promise
• Viral vectors, lipid nanoparticles, and engineered enzymes are characterized by distinct strengths and limitations in enabling in vivo genetic medicines
• Various novel technologies will be reviewed, along with the hurdles that must be overcome to address the challenges of therapeutic delivery
  

9:00 Open Discussion and Key Takeaways

9:30 Course Ends

INSTRUCTORS:

Clifford Steer, MD, Professor of Medicine and, Genetics, Cell Biology, and Development, University of Minnesota Medical School

Clifford J. Steer is a Professor of Medicine and Genetics, Cell Biology, and Development at the University of Minnesota, Minneapolis, MN. He has been active in the field of liver research for more than four decades. In that capacity, he has been a long-standing member of several National Institutes of Health Study Sections. He has been co-editor of a major scientific journal in liver diseases and presently serves on the editorial boards of three journals. Steer’s areas of research over the past decade have included gene therapy, liver regeneration, neurodegeneration and microRNA regulation of gene function. He has published over 300 articles; and has organized and chaired many national and international scientific conferences. In recognition of his work, he was made an inaugural Fellow of the American Association for the Study of Liver Diseases in 2014.

Ross Wilson, PhD, Project Scientist & Principal Investigator, Innovative Genomics Institute, California Institute for Quantitative Biosciences, University of California, Berkeley

Ross Wilson is a principal investigator at the University of California, Berkeley, leading a team at the Innovative Genomics Institute. Dr. Wilson is working to enable widespread clinical use of genome-editing enzymes, which is currently limited by the challenge of delivering enzyme therapeutics to the cells in need of correction. To address this need, the Wilson lab relies on protein/RNA engineering to create safe, effective methods of administration, as well as targeting delivery to specific cells, tissues, or organs. These tools will help genome editing make the leap from the lab to the clinic.

Beau Webber, PhD, Assistant Professor, Department of Pediatrics, Division of Hematology and Oncology, University of Minnesota

Dr. Beau Webber is an Assistant Professor in the Department of Pediatrics, Division of Hematology and Oncology at the University of Minnesota. He graduated from the University of Wisconsin-LaCrosse in 2007 with a BS in Cellular and Molecular Biology and conducted his PhD studies at the University of Minnesota where he studied the embryonic development of hematopoietic stem cells. As a postdoctoral fellow in the Hematology, Oncology, and Transplantation training program at the University of Minnesota, Dr. Webber developed advanced strategies for genetic modification of human lymphohematopoietic and pluripotent stem cells for cancer immunotherapy and targeted correction of disease causing genetic mutations. Dr. Webber joined the Department of Pediatrics Faculty as an Assistant Professor in 2017. The Webber lab is focused on synergizing genome engineering, stem cell biology, and adoptive cellular therapy to develop novel treatments for inherited diseases and cancer. Research projects in the lab currently fall into two broad areas: translational application of genome engineering to develop improved cell-based immune and gene therapies, and the development of “bottom-up” cancer models using human pluripotent stem cells. 

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