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: Benjamin Kleinstiver, John Doench, Kiran Musunuru, Luca Pinello, Becca Cottman, Jonathan Yee-Ting Hsu
Course Director: Keith Joung (
Curriculum Fellow: Kale Hartmann, 

Session Dates and Times
First Session: Thursday, November 7 2019; 9:00am - 12:00pm
First Session Location: Cannon Room
Second Session Date: Tuesday, November 12 2019; 1:00 - 5:00pm
Second Session Location: For registered participants only


Click here to register for this course

*Note: the second session of this nanocourse is full, so you will be placed on the waitlist if you register for it. You are still welcome to attend the first session on November 7th.