Rational Selection of CRISPR-Cas9 Guide RNAs for Homology-Directed Genome Editing

Mol Ther. 2021 Mar 3;29(3):1057-1069. doi: 10.1016/j.ymthe.2020.10.006. Epub 2020 Oct 14.

Abstract

Homology-directed repair (HDR) of a DNA break allows copying of genetic material from an exogenous DNA template and is frequently exploited in CRISPR-Cas9 genome editing. However, HDR is in competition with other DNA repair pathways, including non-homologous end joining (NHEJ) and microhomology-mediated end joining (MMEJ), and the efficiency of HDR outcomes is not predictable. Consequently, to optimize HDR editing, panels of CRISPR-Cas9 guide RNAs (gRNAs) and matched homology templates must be evaluated. We report here that CRISPR-Cas9 indel signatures can instead be used to identify gRNAs that maximize HDR outcomes. Specifically, we show that the frequency of deletions resulting from MMEJ repair, characterized as deletions greater than or equal to 3 bp, better predicts HDR frequency than consideration of total indel frequency. We further demonstrate that tools that predict gRNA indel signatures can be repurposed to identify gRNAs to promote HDR. Finally, by comparing indels generated by S. aureus and S. pyogenes Cas9 targeted to the same site, we add to the growing body of data that the targeted DNA sequence is a major factor governing genome editing outcomes.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • CRISPR-Associated Protein 9 / genetics
  • CRISPR-Associated Protein 9 / metabolism*
  • CRISPR-Cas Systems*
  • DNA Breaks, Double-Stranded
  • DNA End-Joining Repair*
  • Gene Editing*
  • HEK293 Cells
  • Humans
  • INDEL Mutation*
  • K562 Cells
  • RNA, Guide, CRISPR-Cas Systems / genetics*
  • Recombinational DNA Repair*

Substances

  • RNA, Guide, CRISPR-Cas Systems
  • CRISPR-Associated Protein 9
  • Cas9 endonuclease Streptococcus pyogenes