The 1st Tsuneko & Reiji Okazaki Award 2015 Winner

Development and Applications of CRISPR-Cas9 for Genome Editing

Feng Zhang
Core Faculty Member, Broad Institute of MIT and Harvard;
Investigator, McGovern Institute;
W.M. Keck Career Development Professor in Biomedical Engineering, MIT

The Cas9 endonuclease from the microbial adaptive immune system CRISPR can be easily programmed to bind or cleave specific DNA sequence using a short RNA guide. Cas9 is enabling the generation of more realistic disease models and is broadening the number of genetically-tractable organisms that can be used to study a variety of biological processes. The Cas9 nuclease can also be modified to modulate transcription and alter epigenetic states in living cells. In this presentation we will look at the latest developments including structure-guided engineering of Cas9 to achieve robust genome modulation and inducible genome editing, and metagenomic prospecting to identify efficient Cas9 orthologs. We will also describe applications of the Cas9 nuclease for understanding the gene functions in the nervous system and disease processes. The expanding Cas9 toolbox is enabling a broad range of applications in basic research as well as medicine.

Award Lecture