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Nat Biotechnol. 2017 May;35(5):435-437. doi: 10.1038/nbt.3816. Epub 2017 Feb 27.

Highly efficient RNA-guided base editing in mouse embryos.

Kim K1, Ryu SM1,2, Kim ST1, Baek G1, Kim D2, Lim K1,2, Chung E1,2, Kim S1,2, Kim JS1,2.

Author information

1
Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.
2
Department of Chemistry, Seoul National University, Seoul, Republic of Korea.

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.

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PMID:
28244995
DOI:
10.1038/nbt.3816
[Indexed for MEDLINE]

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