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

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

Course Topics
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.

Associate Director

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.

Key:

Complete
Failed
Available
Locked
Background
Open to view video.
Open to view video. In this module we provide a brief background on genes, genomes, and laboratory approaches for profiling gene function. We then present an overview of genome editing technologies with an emphasis on CRISPR-based applications. Lastly, we highlight recent developments in CRISPR-based therapeutics.
Background Quiz
2 Questions  |  Unlimited attempts  |  2/3 points to pass
2 Questions  |  Unlimited attempts  |  2/3 points to pass
Knockout Workflow
Open to view video.
Open to view video. A step-by-step guide to the application of CRISPR/Cas genome editing to create gene knockouts in mammalian cell lines. This module will cover key considerations in gene disruption workflows and what tools are best to achieve success.
Knockout Workflow Quiz
5 Questions  |  Unlimited attempts  |  4/6 points to pass
5 Questions  |  Unlimited attempts  |  4/6 points to pass
Pooled CRISPR Screening
Open to view video.
Open to view video. In this module we cover the fundamentals of large-scale pooled genetic screening with CRISPR/Cas9 technology. We then walk through a series of applications for pooled CRISPR screens.
Pooled CRISPR Screening Quiz
2 Questions  |  Unlimited attempts  |  2/2 points to pass
2 Questions  |  Unlimited attempts  |  2/2 points to pass
Arrayed Screening
Open to view video.
Open to view video. With the long history of plate-based functional genomics in the form of cDNA and siRNA library screening, arrayed CRISPR/Cas offers a complementary methodology that utilizes similar workflows. This module covers the basics of library selection and up-to-date considerations in using arrayed CRISPR/Cas in cell-based screens.
Arrayed Screening Quiz
4 Questions  |  Unlimited attempts  |  3/4 points to pass
4 Questions  |  Unlimited attempts  |  3/4 points to pass
Genome Editing Applications in Drug Discovery and Target Validation Participant Evaluation
6 Questions
6 Questions Thank you for participation in the SLAS Genome Editing Applications in Drug Discovery and Target Validation course. Your candid feedback on this new program is very important. Please take a few minutes to help us make our micro-credential program valuable for all. The topics for the Genome Editing Applications in Drug Discovery and Target Validation course were: 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
Genome Editing Applications in Drug Discovery and Target Validation Certificate
No credits available  |  Certificate available
No credits available  |  Certificate available