SLAS2016 Short Courses

Gene Editing for Drug Discovery (Half-Day; NEW!)

We propose a half-day short course to serve as an introduction to genome engineering applications in drug discovery with an emphasis on the rapidly developing CRISPR/Cas9 technology platform. Given the pace that genome editing tools are reshaping what is possible within the biological sciences, it is timely to survey their proven and potential impact on the process of drug discovery. This course will introduce the audience to the exciting possibilities of what can be achieved with genome editing, the current limitations, and the fundamentals of how to apply these technologies to enhance the pursuit of novel therapeutics.

Who Should Attend

Our target audience are individuals with a general interest in new technologies that impact the process of assay design, development, pre-clinical drug discovery. This includes the implementation of the most recent genome editing tools for endogenous genetic manipulation, model development, and the frontiers of functional genomic (perturbational) screening as part of the discovery process.

How You'll Benefit From This Course

We aim to give the audience a broad, practical perspective on CRISPR/Cas9 technologies so that they understand: 1) the range of state-of-the-art tools; 2) how these tools work; and 3) the key applications associated with genome editing. By the end, they should be able to think about how these technologies may apply to their biological problems to take the next steps towards utilizing them effectively.

Course Topics

1. Overview of the CRISPR/Cas9 system — We will review the basics of CRISPR/Cas9 technology, its bacterial origins, and its rapid implementation to mammalian genetic engineering system.
2. A practical approach to genome editing applications in drug discovery — Provide a user-oriented overview of the considerations for incorporating CRISPR/Cas9 technology in manipulating gene expression and altering genomic DNA sequences. This will include a discussion of the practical aspects of such approaches, such as the formats of the necessary components and their delivery into cells of interest.
3. The exciting frontier of CRISPR/Cas9 in functional genomics and large-scale screening - We will discuss the application of CRISPR technology to genetic screens, that is, using CRISPR reagents as perturbational tools to uncover genes involved in biological processes. This section will examine the range of CRISPR-based tools, such as CRISPR for knockout screening and CRISPRa approaches, as well as provide a comparison to other tools, such as RNAi.

Course Instructors:

John Doench, Broad Institute John G. Doench is the Associate Director of the Genetic Perturbation Platform at the Broad Institute. He develops and applies the latest approaches in functional genomics, including RNAi, ORF, and CRISPR technologies, to understand the function of genes and how gene dysfunction leads to disease. John collaborates with researchers across the Broad, the Boston community, and the world to develop faithful biological models and execute genetic screens.

Samuel Hasson, Pfizer Sam Hasson is a Principal Investigator and Lab Head in Pfizer Neuroscience (Cambridge, Massachusetts). His lab focuses on the application of genome editing and high content analysis technologies to validate and develop novel targets in neurodegenerative disease. A major goal of his work is to identify modulators of complex phenotypes in areas such as neuroinflammation and mitochondrial health by utilizing innovative assay design strategies.

This short course is sponsored by