CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein

Previous works using human tripronuclear zygotes suggested that the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system could be a tool in correcting disease-causing mutations. However, whether this system was applicable in normal human (dual pronuclear, 2PN) zygotes was un...

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Bibliographic Details
Published in:Molecular genetics and genomics : MGG 2017-06, Vol.292 (3), p.525-533
Main Authors: Tang, Lichun, Zeng, Yanting, Du, Hongzi, Gong, Mengmeng, Peng, Jin, Zhang, Buxi, Lei, Ming, Zhao, Fang, Wang, Weihua, Li, Xiaowei, Liu, Jianqiao
Format: Article
Language:English
Subjects:
Animal Genetics and Genomics
Base Sequence
Biochemistry
Biomedical and Life Sciences
Clustered Regularly Interspaced Short Palindromic Repeats - genetics
CRISPR
Embryos
Endonucleases - genetics
Gene Editing - methods
Genetic Diseases, Inborn - genetics
Genetic Diseases, Inborn - therapy
Genome editing
Genome, Human - genetics
Glucosephosphate dehydrogenase
Glucosephosphate Dehydrogenase - genetics
Homeodomain Proteins - genetics
Homologous recombination
Homology
Human Genetics
Humans
Life Sciences
Microbial Genetics and Genomics
Mutation
NIMA-Related Kinase 1 - genetics
Original Article
Plant Genetics and Genomics
Zygote - growth & development
Zygotes
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Summary:Previous works using human tripronuclear zygotes suggested that the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system could be a tool in correcting disease-causing mutations. However, whether this system was applicable in normal human (dual pronuclear, 2PN) zygotes was unclear. Here we demonstrate that CRISPR/Cas9 is also effective as a gene-editing tool in human 2PN zygotes. By injection of Cas9 protein complexed with the appropriate sgRNAs and homology donors into one-cell human embryos, we demonstrated efficient homologous recombination-mediated correction of point mutations in HBB and G6PD . However, our results also reveal limitations of this correction procedure and highlight the need for further research.
ISSN:1617-4615
1617-4623
DOI:10.1007/s00438-017-1299-z