Journal
NATURE REVIEWS DRUG DISCOVERY
Volume 16, Issue 2, Pages 89-100Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nrd.2016.238
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Funding
- US National Institutes of Health from the National Institute of General Medical Sciences (NIGMS) [K99GM118909]
- Li Ka Shing Foundation
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The recent development of CRISPR-Cas systems as easily accessible and programmable tools for genome editing and regulation is spurring a revolution in biology. Paired with the rapid expansion of reference and personalized genomic sequence information, technologies based on CRISPR-Cas are enabling nearly unlimited genetic manipulation, even in previously difficult contexts, including human cells. Although much attention has focused on the potential of CRISPR-Cas to cure Mendelian diseases, the technology also holds promise to transform the development of therapies to treat complex heritable and somatic disorders. In this Review, we discuss how CRISPR-Cas can affect the next generation of drugs by accelerating the identification and validation of high-value targets, uncovering high-confidence biomarkers and developing differentiated breakthrough therapies. We focus on the promises, pitfalls and hurdles of this revolutionary gene-editing technology, discuss key aspects of different CRISPR-Cas screening platforms and offer our perspectives on the best practices in genome engineering.
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