Highly efficient RNA-guided base editing in mouse embryos

被引：320

作者：

Kim, Kyoungmi ^{[1
]}

Ryu, Seuk-Min ^{[1
,2
]}

Kim, Sang-Tae ^{[1
]}

Baek, Gayoung ^{[1
]}

Kim, Daesik ^{[2
]}

Lim, Kayeong ^{[1
,2
]}

Chung, Eugene ^{[1
,2
]}

Kim, Sunghyun ^{[1
,2
]}

Kim, Jin-Soo ^{[1
,2
]}

机构：

[1] Inst for Basic Sci Korea, Ctr Genome Engn, Seoul, South Korea

[2] Seoul Natl Univ, Dept Chem, Seoul, South Korea

来源：

NATURE BIOTECHNOLOGY | 2017年 / 35卷 / 05期

关键词：

HUMAN-CELLS; CAS9; ENDONUCLEASES; MICE; SPECIFICITY; MUTAGENESIS; NICKASES; KNOCKOUT;

D O I：

10.1038/nbt.3816

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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.

引用

页码：435 / +

页数：4

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