Towards a toolbox for mammalian gene editing: Removing off-target activity and protein interactions of Cas9

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In the past 30 years, molecular genetics research has resulted in unprecedented breakthroughs in sequencing and annotating the mammalian genome. One of the main challenges now facing researchers is how to efficiently assign function to each gene. While researchers typically analyze phenotypic changes associated with gene loss-of-function, classical techniques for performing gene knockout in mammalian organisms are slow, laborious, and costly. Recently, DNA sequence-specific nucleases (SSNs) such as TALENs and CRISPR/Cas9 have emerged as tools that are poised to revolutionize this process. An over-arching goal of my lab is to develop robust technology for genetically altering mammalian organisms with high precision. Through a combination of molecular evolution and proteomics approaches, this program will develop a gene editing toolbox for mammals that will realize this possibility.