SC12: Emerging Applications for Gene Editing, Base Editing and RNA Editing
TUESDAY, MAY 5 | DINNER, 6:00 – 8:30 PM
ABOUT THIS COURSE:
While the challenges and risks associated with oligonucleotide therapies still remain, there is a new and better understanding of how DNA and RNA 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, base editing and RNA 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.
COURSE AGENDA:
5:30 Welcome Remarks and Speaker Introductions
5:40 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
6:30 Gene-edited Cell-based Therapies
Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital
- Sources of therapeutic cells (Stem cells, T cells, NK cells and Cancer cells)
- Novel engineered viral vectors and their engineering capabilities
- Role of CRISPR gene editing in creating therapeutic cells
- Applications of gene edited cells in pre-clinical models
- Clinical translation and safety aspects of engineered cell therapies
7:15 Coffee and Dessert Break
7:30 Harnessing Novel CRISPR Systems for Transcriptome Engineering
Jonathan Gootenberg, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT
Omar Abudayyeh, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT
- Many novel DNA and RNA targeting CRISPR systems can be found from bacterial genome mining
- Cas13 is a programmable RNA targeting system that can be used for RNA base editing
- REPAIR and RESCUE Cas13 RNA editors are capable of correcting disease-relevant mutations in RNA
8:15 Q&A with attendees
8:30 End of Course
INSTRUCTOR BIOGRAPHIES:
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.
Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital
Khalid Shah, MS, PhD is the Director of the Center for Stem Cell Therapeutics and Imaging and the Center of Excellence in Biomedicine at Brigham and Women’s Hospital (BWH). He is also the Vice Chair of Research for the Department of Neurosurgery
at BWH and a Principal Faculty at Harvard Stem Cell Institute in Boston. Dr. Shah and his team have pioneered major developments in the cell therapy field, successfully developing experimental models to understand basic cancer biology and therapeutic
cells for cancer. Recently, Dr. Shah’s laboratory has reverse engineered cancer cells using CRISPR/Cas9 technology and utilized them as therapeutics to treat cancer. He has founded 2 biotech companies, AMASA Therapeutics and ALIM Therapeutics,
whose main objective is the clinical translation of therapeutic stem cells in cancer patients.
Jonathan Gootenberg, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT
Jonathan Gootenberg draws from fundamental microbiology to engineer new molecular tools. These tools, including the popular genome editing system CRISPR, allow for unprecedented manipulation and profiling of cellular states in the body, and have multiple
applications in basic science, diagnostics, and therapeutics. Dr. Gootenberg uses gene editing, gene delivery, and cellular profiling methods to understand the changes that occur in the brain and other organs during aging, with the goal of generating
new therapies for degenerative disease. Dr. Gootenberg earned his bachelor’s degree in mathematics and biological engineering at MIT and received his PhD in Systems Biology from Harvard University, during which he conducted research with Aviv
Regev and Feng Zhang at the McGovern Institute and Broad Institute of MIT and Harvard. During his PhD, Gootenberg focused on the development of molecular technologies for treating and sensing disease states, crossing disciplines by utilizing novel
computational techniques, microbiology, biochemistry, and molecular biology to uncover new CRISPR tools, including Cas12 and Cas13. He and his co-authors developed Cas13 into a toolbox with uses in fundamental research, therapeutics, and diagnostics.
These applications include RNA knockdown, imaging, the base editing platform REPAIR, and the sensitive, specific, and portable diagnostic platform SHERLOCK. He is one of the first members of the McGovern Institute Fellows program, which supports the
transition to independent research for exceptional recent PhD graduates.
Omar Abudayyeh, PhD, McGovern Fellow, McGovern Institute for Brain Research, MIT
Omar Abudayyeh, Ph.D., is a McGovern Institute Fellow at the Massachusetts Institute of Technology where he leads a lab on exploring microbial diversity for new biotechnological tools related to genome editing and gene delivery. He previously was at Harvard
Medical School and the Harvard-MIT Health Sciences and Technology program as a graduate student. He completed his doctoral work in Feng Zhang’s lab at the Broad Institute of MIT and Harvard, where his research centered on novel CRISPR enzymes
for applications in genome editing, therapeutics, and diagnostics. Dr. Abudayyeh’s work focused on uncovering novel CRISPR enzymes beyond Cas9 for biotechnological applications. He co-led the discovery and characterization of multiple landmark
pieces of work, including the characterization of Cpf1 for novel genome editing applications and the first single-protein RNA-guided RNA-targeting enzyme C2c2/Cas13. His follow-up work on C2c2/Cas13 biology led to the development of SHERLOCK technology,
and a new set of tools for precise editing of transcripts and visualizing them in mammalian cells with potential for RNA therapeutics. In recognition of his technology developments, Dr. Abudayyeh was recognized as 2018 Forbes 30 under 30 in Science
and Health Care and Business Insider 30 under 30. Dr. Abudayyeh graduated from MIT in 2012 with a B.S. in mechanical engineering and biological engineering, where he was a Henry Ford II Scholar and a Barry M. Goldwater Scholar.