Efficient targeted integration directed by short homology in zebrafish and mammalian cells

Elife. 2020 May 15:9:e53968. doi: 10.7554/eLife.53968.

Abstract

Efficient precision genome engineering requires high frequency and specificity of integration at the genomic target site. Here, we describe a set of resources to streamline reporter gene knock-ins in zebrafish and demonstrate the broader utility of the method in mammalian cells. Our approach uses short homology of 24-48 bp to drive targeted integration of DNA reporter cassettes by homology-mediated end joining (HMEJ) at high frequency at a double strand break in the targeted gene. Our vector series, pGTag (plasmids for Gene Tagging), contains reporters flanked by a universal CRISPR sgRNA sequence which enables in vivo liberation of the homology arms. We observed high rates of germline transmission (22-100%) for targeted knock-ins at eight zebrafish loci and efficient integration at safe harbor loci in porcine and human cells. Our system provides a straightforward and cost-effective approach for high efficiency gene targeting applications in CRISPR and TALEN compatible systems.

Keywords: CRISPR/Cas9; developmental biology; end joining; genetics; genomics; human; knock-in; pig fibroblasts; targeted integration; zebrafish.

Publication types

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

MeSH terms

  • Animals
  • Animals, Genetically Modified
  • CRISPR-Associated Proteins / genetics*
  • CRISPR-Associated Proteins / metabolism
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats*
  • Fibroblasts / metabolism
  • Gene Expression Regulation
  • Gene Knock-In Techniques*
  • Genes, Reporter*
  • Green Fluorescent Proteins / genetics*
  • Green Fluorescent Proteins / metabolism
  • Humans
  • K562 Cells
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / genetics
  • Leukemia, Myelogenous, Chronic, BCR-ABL Positive / metabolism
  • RNA, Guide, CRISPR-Cas Systems / genetics
  • RNA, Guide, CRISPR-Cas Systems / metabolism
  • Recombinational DNA Repair
  • Sequence Homology, Nucleic Acid
  • Sus scrofa
  • Transcription Activator-Like Effector Nucleases / genetics*
  • Transcription Activator-Like Effector Nucleases / metabolism
  • Zebrafish / genetics*

Substances

  • CRISPR-Associated Proteins
  • RNA, Guide, CRISPR-Cas Systems
  • Green Fluorescent Proteins
  • Transcription Activator-Like Effector Nucleases

Associated data

  • Dryad/10.5061/dryad.m63xsj3zc