CRISPR-Cpf1 mediates efficient homology-directed repair and temperature-controlled genome editing

被引:234
作者
Moreno-Mateos, Miguel A. [1 ]
Fernandez, Juan P. [1 ]
Rouet, Romain [2 ,3 ]
Vejnar, Charles E. [1 ]
Lane, Maura A. [1 ,4 ]
Mis, Emily [1 ,4 ]
Khokha, Mustafa K. [1 ,4 ]
Doudna, Jennifer A. [2 ,3 ,5 ,6 ,7 ,8 ]
Giraldez, Antonio J. [1 ,9 ,10 ]
机构
[1] Yale Univ, Dept Genet, Sch Med, New Haven, CT 06510 USA
[2] Univ Calif Berkeley, Dept Mol & Cell Biol, 229 Stanley Hall, Berkeley, CA 94720 USA
[3] Univ Calif Berkeley, Calif Inst Quantitat Biosci, Berkeley, CA 94720 USA
[4] Yale Univ, Sch Med, Dept Pediat, New Haven, CT 06520 USA
[5] Univ Calif Berkeley, Dept Chem, Berkeley, CA 94720 USA
[6] Univ Calif Berkeley, Innovat Genom Initiat, Berkeley, CA 94720 USA
[7] Lawrence Berkeley Natl Lab, MBIB Div, Berkeley, CA 94720 USA
[8] Univ Calif Berkeley, Howard Hughes Med Inst, Berkeley, CA 94720 USA
[9] Yale Univ, Yale Stem Cell Ctr, Sch Med, New Haven, CT 06510 USA
[10] Yale Univ, Yale Canc Ctr, Sch Med, New Haven, CT 06510 USA
基金
美国国家卫生研究院; 瑞士国家科学基金会; 英国医学研究理事会;
关键词
TARGETED MUTAGENESIS; MESSENGER-RNA; CPF1; DNA; MICE; SPECIFICITIES; ENDONUCLEASES;
D O I
10.1038/s41467-017-01836-2
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
070301 [无机化学]; 070403 [天体物理学]; 070507 [自然资源与国土空间规划学]; 090105 [作物生产系统与生态工程];
摘要
Cpf1 is a novel class of CRISPR-Cas DNA endonucleases, with a wide range of activity across different eukaryotic systems. Yet, the underlying determinants of this variability are poorly understood. Here, we demonstrate that LbCpf1, but not AsCpf1, ribonucleoprotein complexes allow efficient mutagenesis in zebrafish and Xenopus. We show that temperature modulates Cpf1 activity by controlling its ability to access genomic DNA. This effect is stronger on AsCpf1, explaining its lower efficiency in ectothermic organisms. We capitalize on this property to show that temporal control of the temperature allows post-translational modulation of Cpf1-mediated genome editing. Finally, we determine that LbCpf1 significantly increases homology-directed repair in zebrafish, improving current approaches for targeted DNA integration in the genome. Together, we provide a molecular understanding of Cpf1 activity in vivo and establish Cpf1 as an efficient and inducible genome engineering tool across ectothermic species.
引用
收藏
页数:9
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