Highly efficient RNA-guided base editing in mouse embryos

  • Nat Biotechnol. 2017 May;35(5):435-437. doi: 10.1038/nbt.3816.
Kyoungmi Kim  1 ,  Seuk-Min Ryu  1  2 ,  Sang-Tae Kim  1 ,  Gayoung Baek  1 ,  Daesik Kim  2 ,  Kayeong Lim  1  2 ,  Eugene Chung  1  2 ,  Sunghyun Kim  1  2 ,  Jin-Soo Kim  1  2
Affiliations
  • 1. Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.
  • 2. Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
PMID: 28244995 DOI: 10.1038/nbt.3816
Abstract

Base editors (BEs) composed of a cytidine deaminase fused to CRISPR-Cas9 convert cytidine to uridine, leading to single-base-pair substitutions in eukaryotic cells. We delivered BE mRNA or ribonucleoproteins targeting the Dmd or Tyr gene via electroporation or microinjection into mouse zygotes. F0 mice showed nonsense mutations with an efficiency of 44-57% and allelic frequencies of up to 100%, demonstrating an efficient method to generate mice with targeted point mutations.