Modulating CRISPR/Cas9 genome-editing activity by small molecules

Siwei Chen, Deng Chen, Bin Liu, Hidde J Haisma*

*Bijbehorende auteur voor dit werk

Onderzoeksoutputpeer review

11 Citaten (Scopus)
127 Downloads (Pure)


Clustered 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.

Originele taal-2English
Pagina's (van-tot)951-966
Aantal pagina's16
TijdschriftDrug Discovery Today
Nummer van het tijdschrift4
Vroegere onlinedatum22-nov.-2021
StatusPublished - apr.-2022

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