Efficient and error-free fluorescent gene tagging in human organoids without double-strand DNA cleavage

PLoS Biol. 2022 Jan 28;20(1):e3001527. doi: 10.1371/journal.pbio.3001527. eCollection 2022 Jan.

Abstract

CRISPR-associated nucleases are powerful tools for precise genome editing of model systems, including human organoids. Current methods describing fluorescent gene tagging in organoids rely on the generation of DNA double-strand breaks (DSBs) to stimulate homology-directed repair (HDR) or non-homologous end joining (NHEJ)-mediated integration of the desired knock-in. A major downside associated with DSB-mediated genome editing is the required clonal selection and expansion of candidate organoids to verify the genomic integrity of the targeted locus and to confirm the absence of off-target indels. By contrast, concurrent nicking of the genomic locus and targeting vector, known as in-trans paired nicking (ITPN), stimulates efficient HDR-mediated genome editing to generate large knock-ins without introducing DSBs. Here, we show that ITPN allows for fast, highly efficient, and indel-free fluorescent gene tagging in human normal and cancer organoids. Highlighting the ease and efficiency of ITPN, we generate triple fluorescent knock-in organoids where 3 genomic loci were simultaneously modified in a single round of targeting. In addition, we generated model systems with allele-specific readouts by differentially modifying maternal and paternal alleles in one step. ITPN using our palette of targeting vectors, publicly available from Addgene, is ideally suited for generating error-free heterozygous knock-ins in human organoids.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles
  • Base Sequence
  • CRISPR-Associated Protein 9 / genetics
  • CRISPR-Associated Protein 9 / metabolism
  • Colon / cytology
  • Colon / metabolism
  • DNA / genetics*
  • DNA / metabolism
  • DNA End-Joining Repair
  • Deoxyribonuclease I / genetics
  • Deoxyribonuclease I / metabolism*
  • Electroporation / methods
  • Epithelial Cells / cytology
  • Epithelial Cells / metabolism
  • Fluorescent Dyes / chemistry
  • Fluorescent Dyes / metabolism
  • Gene Knock-In Techniques
  • Genetic Loci*
  • Genetic Vectors
  • Genome, Human
  • Heterozygote
  • Humans
  • Organoids / cytology
  • Organoids / metabolism*
  • Recombinational DNA Repair*
  • Staining and Labeling / methods*

Substances

  • Fluorescent Dyes
  • DNA
  • CRISPR-Associated Protein 9
  • Deoxyribonuclease I

Grants and funding

This work is part of the Oncode Institute, which is partly financed by the Dutch Cancer Society. HGJS received European Research Council (ERC) starting grant (IntratumoralNiche), project number 803608 (https://erc.europa.eu/funding/starting-grants) and NWO TOP. YB was supported by a strategic alliance between University of Twente and UMC Utrecht on Advanced Biomanufacturing (to LWMMT and HJGS). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.