Lawrence Berkeley National Laboratory
Cornerstones of CRISPR-Cas in drug discovery and therapy.
- Author(s): Fellmann, Christof
- Gowen, Benjamin G
- Lin, Pei-Chun
- Doudna, Jennifer A
- Corn, Jacob E
- et al.
Published Web Locationhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC5459481/
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.