CRISPR Gene Editing for Studying Disease Pathology and Treating Human Disease





Clustered regularly interspaced short palindromic repeat (CRISPR) RNAs and their CRISPR-associated (Cas) proteins are an important part of adaptive immune systems in many prokaryotes. CRISPR-Cas systems function as RNA-directed endonucleases that can target nucleic acids in a sequence-specific manner and are now widely used as genome editing tools. In this course, we will provide lectures covering: an introduction to genome editing and cutting-edge improvements to CRISPR-Cas systems; a review of bioinformatics tools for guide RNA design and analysis of CRISPR-Cas data; and an overview of ongoing and potential therapeutic applications of genome-editing nucleases. The course will also include a practical lab-based workshop for registered students in which participants will learn how to design guide RNAs and how to quantify nuclease-induced mutations in any cell or organism using sequencing-based assays.

Assignment: Complete analysis of a sequencing-based experiment designed to assess mutation frequency induced by a CRISPR-Cas nuclease at an endogenous human gene target site.

Course Instructors: Luca Pinello, John Doench, Morgan Maeder, Becca Cottman, and Ben Kleinstiver
Course Director: Keith Joung (
Curriculum Fellow: Rachel Wright,

Session Dates and Times
First Session: Monday, November 5 2018; 1:00 - 4:00pm
First Session Location: Cannon Room
Second Session Date: Wednesday, November 14 2018; 1:30 - 5:30pm
Second Session Location: For registered participants only