Genome Editing Applications in Drug Discovery and Target Validation

February 25 - March 1, 2023

San Diego, CA, USA

Genome Editing Applications in Drug Discovery and Target Validation

Genome editing tools such as CRISPR-Cas9 are reshaping what is possible within biological sciences. This course will introduce the state-of-the-art in genome editing, the current limitations and the fundamentals of how to apply these technologies to enhance the pursuit of 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

Course Benefits

Up-to-date information on the tools, methods and applications of genome editing
Workflows for typical experiments and practical use cases
Case studies of how genome editing is enabling discovery research

Course Topics

Genome Engineering Concepts
Gene Editing in 2022: Workflows for Creating Knockouts and Knock in Cell Lines
CRISPR-Cas9 and Beyond: Tools of the Trade
Applications of Genome Editing in Functional Genomics
A Practical Guide to Using Genome Editing Tools in Genetic Screens

Instructors

Scott T. Younger
Children’s Mercy Research Institute, Children’s Mercy Kansas City

Scott Younger is the Director of Disease Gene Engineering within the Genomic Medicine Center 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
Voyager Therapeutics

Sam Hasson is currently an Associate Director at Voyager Therapeutics (Cambridge, Massachusetts). A major aim of his work is to develop novel applications of AAV-based gene therapy in the CNS to transform the treatment of unmet medical needs. Prior to joining Voyager in 2020, Sam led groups within Amgen Neuroscience and Pfizer Neuroscience with a focus on the deconvolution of human disease genetics for drug target selection, employing genome editing technologies to enable this process. As a postdoc, Sam trained with Richard Youle and Jim Inglese at the National Institutes of Health.