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Nat Rev Drug Discov. 2017 Feb;16(2):89-100. doi: 10.1038/nrd.2016.238. Epub 2016 Dec 23.

Cornerstones of CRISPR-Cas in drug discovery and therapy.

Fellmann C1, Gowen BG1,2, Lin PC1,2,3, Doudna JA1,4,5,6,7, Corn JE1,2.

Author information

1
Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, California 94720, USA.
2
Innovative Genomics Institute, University of California, Berkeley, Berkeley, California 94720, USA.
3
Present address: Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, California 94158, USA.
4
Department of Chemistry, University of California, Berkeley, Berkeley, California 94720, USA.
5
Howard Hughes Medical Institute, University of California, Berkeley, Berkeley, California 94720, USA.
6
Li Ka Shing Biomedical and Health Sciences Center, University of California, Berkeley, Berkeley, California 94720, USA.
7
MBIB Division, Lawrence Berkeley National Laboratory, Berkeley, California 94720, USA.

Abstract

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.

PMID:
28008168
PMCID:
PMC5459481
DOI:
10.1038/nrd.2016.238
[Indexed for MEDLINE]
Free PMC Article

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