Effect of the Phosphoryl Guanidine Modification in Chimeric DNA-RNA crRNAs on the Activity of the CRISPR-Cas9 System In Vitro

ACS Chem Biol. 2024 Jun 21;19(6):1311-1319. doi: 10.1021/acschembio.4c00147. Epub 2024 May 30.

Abstract

Currently, the CRISPR-Cas9 system serves as a prevalent tool for genome editing and gene expression regulation. Its therapeutic application is limited by off-target effects that can affect genomic integrity through nonspecific, undesirable changes in the genome. Various strategies have been explored to mitigate the off-target effects. Many approaches focus on modifying components of the system, namely, Cas9 and guide RNAs, to enhance specificity. However, a common challenge is that methods aiming to increase specificity often result in a significant reduction in the editing efficiency. Here, we introduce a novel approach to modifying crRNA to balance CRISPR-Cas9 specificity and efficiency. Our approach involves incorporating nucleoside modifications, such as replacing ribo- to deoxyribonucleosides and backbone modifications, using phosphoryl guanidine groups, specifically 1,3-dimethylimidazolidin-2-ylidene phosphoramidate. In this case, within the first 10 nucleotides from the 5' crRNA end, phosphodiester bonds are substituted with phosphoryl guanidine groups. We demonstrate that crRNAs containing a combination of deoxyribonucleosides and single or multiple phosphoryl guanidine groups facilitate the modulation of CRISPR-Cas9 system activity while improving its specificity in vitro.

MeSH terms

  • CRISPR-Cas Systems*
  • DNA* / chemistry
  • DNA* / metabolism
  • Gene Editing* / methods
  • Guanidine* / chemistry
  • Guanidines / chemistry
  • Humans
  • RNA / chemistry
  • RNA / metabolism
  • RNA, Guide, CRISPR-Cas Systems* / chemistry
  • RNA, Guide, CRISPR-Cas Systems* / genetics
  • RNA, Guide, CRISPR-Cas Systems* / metabolism

Substances

  • DNA
  • Guanidine
  • RNA, Guide, CRISPR-Cas Systems
  • RNA
  • Guanidines