Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases

Young Hoon Sung, Jong Min Kim, Hyun Taek Kim, Jaehoon Lee, Jisun Jeon, Young Jin, Jung Hwa Choi, Young Ho Ban, Sang Jun Ha, Cheol Hee Kim, Han Woong Lee, Jin Soo Kim

Research output: Contribution to journalArticlepeer-review

219 Citations (Scopus)


RNA-guided endonucleases (RGENs), derived from the prokaryotic Type II CRISPR-Cas system, enable targeted genome modification in cells and organisms. Here we describe the establishment of gene-knockout mice and zebrafish by the injection of RGENs as Cas9 protein:guide RNA complexes or Cas9 mRNA plus guide RNA into one-cell-stage embryos of both species. RGENs efficiently generated germline transmittable mutations in up to 93% of newborn mice with minimal toxicity. RGEN-induced mutations in the mouse Prkdc gene that encodes an enzyme critical for DNA double-strand break repair resulted in immunodeficiency both in F0 and F1 mice. We propose that RGEN-mediated mutagenesis in animals will greatly expedite the creation of genetically engineered model organisms, accelerating functional genomic research.

Original languageEnglish
Pages (from-to)125-131
Number of pages7
JournalGenome Research
Issue number1
Publication statusPublished - 2014 Jan

All Science Journal Classification (ASJC) codes

  • Genetics
  • Genetics(clinical)


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