SC14: A Primer to Gene Editing: Tools and Applications

Wednesday, September 23, 7:00-9:30 pm (Dinner provided.)

The course will help the novice understand the basics of how gene editing works, what tools are available for use and how those tools differ from each other. For the expert, this course will offer details on the CRISPR technology, how to set up CRISPR-based screens and complement it with existing RNAi-based screens using proper analysis and follow-up studies. The instructors will also cover the use of gene editing in drug discovery and disease modeling and best practices for design and workflows when working with other model systems, besides mammalian cells.

Course Instructors:

Genetic Screens with CRISPRs

John Doench, Ph.D., Research Scientist, Broad Institute of Harvard and MIT

• Considerations for assay development - screening strategies unique to CRISPR technology
• Expected performance and comparison to RNAi - on-target activity and specificity
• Proper analysis and follow-up

Comparative Analysis of shRNA and CRISPR/Cas9 based screens

Michael Bassik, Ph.D., Assistant Professor, Department of Genetics, Stanford University

• Identification of drug targets and phenotypes for essential genes
• Screen design/common pitfalls
• Optimized protocols for sample prep, sequencing, and analysis

Gene Editing in Drug Discovery and Disease Modeling

Mi Cai, Ph.D., Senior Scientist, Neuroscience & Pain Research Unit, Pfizer, Inc.

• CRISPR-Cas9 gene editing for drug target validation in mammalian cells
• Generation of cellular and animal disease models with CRISPR-Cas9
• Control of gene expression by CRISPR-Cas9 system

Gene Editing Tips and Tricks: Borrowing From Model Systems

Stephanie Mohr, Ph.D., Department of Genetics, Harvard Medical School

• Algorithms for sgRNA design and evaluation 
• Workflows for cell-based and in vivo knockouts
• Next-generation CRISPR-Cas approaches

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