Genome Editing Applications in Drug Discovery and Target Validation
Genome editing tools such as CRISPR-Cas9 are reshaping what is possible within the biological sciences. This course will provide a focused introduction into some of the most pertinent applications of genome editing in drug discovery and target identification/validation. With practical methods in cell line generation and functional genomic screening, the course provides a perspective on how these technologies can work to enhance the pursuit of new drug targets and therapeutics.
Who Should Attend
Assay and HTS biologists
Those interested in drug discovery technology
Scientists working on functional genomics and genetic screens
Those wanting to learn the basics of CRISPR/Cas9 technology and how it can be practically applied
Up-to-date information on the tools, methods and applications of genome editing
Workflows for typical experiments and practical use cases
Genome engineering concepts
Gene editing in 2020: Workflows to creating knockouts and knock-in cell lines
CRISPR-Cas9 and beyond – tools of the trade
Applications of genome editing in functional genomics
Practical guides to using genome editing tools in genetic screens
Scott T. Younger, Ph.D
Director of Disease Gene Engineering
Children’s Research Institute, Children’s Mercy Kansas City
Scott Younger, Ph.D., is the Director of Disease Gene Engineering within the Center for Pediatric Genomic Medicine at Children's Mercy Kansas City. His laboratory is focused on dissecting the molecular mechanisms through which rare genetic variants identified in patients at Children's Mercy lead to disease. Younger joined Children's Mercy from the Broad Institute of MIT and Harvard where his group worked on the development of new methodologies to expand the utility of CRISPR-based genetic screens. Prior to working at the Broad Institute, he completed his postdoctoral studies at Harvard University as an American Cancer Society Fellow. He holds a Ph.D. in cell and molecular biology from UT Southwestern Medical Center. He also received an M.S. in biotechnology from the University of Texas at San Antonio and a B.S.I. in bioinformatics from Baylor University.
Samuel A. Hasson, Ph.D.
Voyager Therapeutics, Inc.
Sam Hasson is currently an Associate Director at Voyager Therapeutics (Cambridge, Massachusetts). His lab focuses on emerging applications of gene therapy in the central nervous system in addition to expanding the molecular toolbox of gene therapy. Previously, as a lab head within Amgen Neuroscience and Pfizer Neuroscience, Sam has utilized genome editing in the deconvolution of human genetics to enable target validation and selection. A major goal of Sam’s past work in industry has been to identify modulators of neuroimmune phenotypes utilizing innovative assay design strategies such as high content screening. As a postdoc, Sam trained with Richard Youle and Jim Inglese at the National Institutes of Health.
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