Development and Applications of CRISPR-Cas9 for Genome Editing

Feng Zhang


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Duration: 1 h 1 m

Date: March 2, 2015


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. Previously we have demonstrated that the Cas9 nuclease can be successfully engineered to facilitated genome editing by targeting DNA double strand breaks or single strand nicking in mammalian cells. 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. We will also describe applications of the Cas9 nuclease for understanding the gene functions in the nervous system and disease processes.

Further Information:

Dr. Zhang’s lab is focused on using synthetic biology to develop technologies for genome and epigenome engineering to study neurobiology. As a postdoc, he began work on using TAL effectors to control gene transcription. However, given the difficulty of using such tools, he looked to develop new genome-editing tools better suited for his research. In 2011, Zhang studied the scientific literature on the CRISPR system and, synthesizing evidence from existing foundational studies of the natural system, including a study by Sylvain Moineau’s group describing the DNA-cleavage activity of Cas9, he set out to harness the CRISPR system (found only in prokaryotic cells) for genome editing in mammalian/human cells.
Dr. Zhang is a recipient of the NIH Pioneer Award and a Searle Scholar. He was named one of MIT Technology Reviews’s TR35 in 2013. His work on optogenetics and CRISPR has been recognized by a number of awards including: the 2012 Perl-UNC Neuroscience Prize (for optogenetics, shared with Boyden and Deisseroth; the 2014Alan T. Waterman Award (for optogenetics and CRISPR-Cas9), the National Science Foundation’s highest honor that annually recognizes an outstanding researcher under the age of 35; the 2014 Jacob Heskel Gabbay Award in Biotechnology and Medicine (for CRISPR-Cas9, shared with Doudna and Charpentier); and the 2014 Young Investigator Award from the Society for Neuroscience (for optogenetics and CRISPR-Cas9).

Dr. Zhang is a founder of Editas Medicine, a genome-editing company founded by world leaders in the fields of genome editing, protein engineering, and molecular and structural biology, with specific expertise in CRISPR-Cas9 and TALE technologies.

Created: Monday, March 2nd, 2015