MONDAY, MARCH 27 | 6:30-9:30 PM
SC3: CRISPR-BASED GENE EDITING FOR TARGETED THERAPIES


While the challenges and risks associated with oligonucleotide therapies still remain, there is a new and better understanding of how genes can be effectively manipulated and delivered. With the rise of gene editing tools and enhanced knowledge of targeted delivery, these therapeutic modalities are once again being embraced with renewed hope and enthusiasm. This course helps you understand how gene editing – particularly the one enabled by the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas9 system – works, and how it can be used to help develop targeted therapies with good efficacy and delivery.

6:30 Welcome and Opening Remarks

6:45 The Cure to Human (and Non-Human) Disease Through Gene Editing

Clifford Steer, M.D., Professor of Medicine and, Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

  • Evaluating target-based gene editing in the realm of personalized medicine
  • Identifying the appropriate clinical targets
  • Optimizing the strategies required to cure diseases
  • Establishing the safety parameters for gene editing
  • Identifying the translational aspects of preclinical safety assessments
  • Understanding the ethical issues involved in gene editing for human clinical disease

7:30 Coffee Break

7:45 CRISPR/Cas9 Genome Editing Tools: From Mouse Modeling to Potential Therapies

Ciro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals

  • Germline and somatic gene modifications to generate mouse models to study disease
  • New tools to improve precise genome editing
  • A new era for gene therapy (challenges and potential applications)

8:30 Delivery Technologies for Genome Editing

Hao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

  • Barriers to delivery of genome editing components
  • Physical methods and non-viral vectors used for delivery of genome editing biomacromolecules
  • Main types of viruses used for genome editing
  • Manufacturing issues for genome editing therapies

9:15 Open Discussion

9:30 End of Course

Speaker Biographies

Clifford_SteerClifford Steer, M.D., Professor of Medicine and, Genetics, Cell Biology, and Development; Director, Molecular Gastroenterology Program, University of Minnesota Medical School

Clifford J. Steer is a Professor of Medicine and Genetics, Cell Biology, and Development and a senior investigator at the University of Minnesota, Minneapolis, MN. He has been active in the field of liver research for almost 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. He has published his work in top-ranking scientific journals and numerous textbooks. Steer’s areas of research over the last decade have included gene therapy, liver regeneration, and microRNA regulation of gene function. He has published over 250 scientific articles; and has organized and chaired many national and international scientific conferences. In recognition of his work he has received the Holloman Award in Biotechnology in 1998; the Thorne Stroke Award and Department of Medicine Faculty Award for Outstanding Research both in 2004; and the Senior Investigator Award from the University of Minnesota Medical School in 2012. He was elected into the American Society for Clinical Investigation in 1991; and in 2014 was made an inaugural Fellow of the American Association for the Study of Liver Diseases. His work has been written up in newspapers around the world, Time magazine, and in 1998 was featured in the Village Voice. He was most recently appointed as Associate Dean for Faculty Affairs in the Medical School.

Ciro_BonettiCiro Bonetti, Ph.D., Scientist, Regeneron Pharmaceuticals

Ciro Bonetti is currently a Scientist at Regeneron Pharmaceuticals® in New York. He earned his PhD in Medical Genetics at TIGEM, Telethon Institute of Genetics and Medicine in Italy, where he applied and developed gene therapy approaches for recessive and dominant retinal congenital disorders. As American-Italian Cancer Foundation fellow, he moved for his postdoc to Memorial Sloan-Kettering Cancer Center in New York. During this time, he became an expert in a variety of genome editing tools including CRISPR/Cas9, which he has used to understand how non-coding genes affect normal physiology and impact human disease. He also adapted these tools to generate precise somatic mutations in terminally differentiated cells. This will impact not only our ability to create more precise animal models of human disease but will also pave the way to novel therapeutic opportunities.

Hao_YinHao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

Hao Yin performed his postdoctoral training in the laboratories of Robert Langer and Daniel G. Anderson at Massachusetts Institute of Technology. His research has harnessed small interfering RNA and genome editing tools for disease modelling and gene-based therapy. His work has led to the first demonstration that CRISPR could correct a disease mutation in vivo and reverse disease symptoms in adult living animals. Hao received his B.S. in biochemistry at Nanjing University, China, and his Ph.D. in toxicology from the School of Pharmacy, University of Colorado Anschutz Medical Campus, Aurora, USA.