Journal
TRENDS IN PHARMACOLOGICAL SCIENCES
Volume 43, Issue 2, Pages 151-161Publisher
CELL PRESS
DOI: 10.1016/j.tips.2021.10.012
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Funding
- Defense Advanced Research Projects Agency [N66001-17-2-4055]
- National Institutes of Health [R01GM132825, R01GM137606]
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The discovery of CRISPR-Cas systems has revolutionized therapeutic development and treatment approaches, with the ability to rapidly identify drug mechanisms, advance ex vivo therapies, and address limitations in in vivo treatments.
The discovery, only a decade ago, of the genome editing power of clustered regularly interspaced short palindromic repeats (CRISPR)-associated nucleases is already reinventing the therapeutic process, from how new drugs are discovered to novel ways to treat diseases. CRISPR-based screens can aid therapeutic development by quickly identifying a drug's mechanism of action and escape mutants. Additionally, CRISPR-Cas has advanced emerging ex vivo therapeutics, such as cell replacement therapies. However, Cas9 is limited as an in vivo therapeutic due to ineffective delivery, unwanted immune responses, off-target effects, unpredictable repair outcomes, and cellular stress. To address these limitations, controls that inhibit or degrade Cas9, biomolecule-Cas9 conjugates, and base editors have been developed. Herein, we discuss CRISPR-Cas systems that advance both conventional and emerging therapeutics.
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