Modulating CRISPR/Cas9 genome-editing activity by small molecules
UMass Chan AffiliationsRNA Therapeutics Institute
Document TypeJournal Article
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AbstractClustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9)-mediated genome engineering has become a standard procedure for creating genetic and epigenetic changes of DNA molecules in basic biology, biotechnology, and medicine. However, its versatile applications have been hampered by its overall low precise gene modification efficiency and uncontrollable prolonged Cas9 activity. Therefore, overcoming these problems could broaden the therapeutic use of CRISPR/Cas9-based technologies. Here, we review small molecules with the clinical potential to precisely modulate CRISPR/Cas9-mediated genome-editing activity and discuss their mechanisms of action. Based on these data, we suggest that direct-acting small molecules for Cas9 are more suitable for precisely regulating Cas9 activity. These findings provide useful information for the identification of novel small-molecule enhancers and inhibitors of Cas9 and Cas9-associated endonucleases.
SourceChen S, Chen D, Liu B, Haisma HJ. Modulating CRISPR/Cas9 genome-editing activity by small molecules. Drug Discov Today. 2022 Apr;27(4):951-966. doi: 10.1016/j.drudis.2021.11.018. Epub 2021 Nov 22. PMID: 34823004.
Permanent Link to this Itemhttp://hdl.handle.net/20.500.14038/51811
RightsCopyright 2021 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).; Attribution 4.0 International
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Except where otherwise noted, this item's license is described as Copyright 2021 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).