Effective and precise adenine base editing in mouse zygotes

Protein and Cell

Volumn 9, Issue 9, 2018, Pages 808-813

  Liang, Puping ^a   Sun, Hongwei ^a   Zhang, Xiya ^a   Xie, Xiaowei ^a   Zhang, Jinran ^a   Bai, Yaofu ^a   Ouyang, Xueling ^a   Zhi, Shengyao ^a   Xiong, Yuanyan ^a   Ma, Wenbin ^a   Liu, Dan ^c   Huang, Junjiu ^a,b   Songyang, Zhou ^a,b,c  

^a SUN YAT SEN UNIVERSITY   (China)

^b SUN YAT SEN UNIVERSITY   (China)

^c BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; CREATINE KINASE; ADENINE;

ADENINE BASE EDITING; ALLELE; DNA REPLICATION; ENZYME ACTIVITY; GENE MUTATION; GENETIC PROCEDURES; HUMAN; LETTER; MUSCULAR DYSTROPHY; NONHUMAN; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; RNA SPLICING; SANGER SEQUENCING; ZYGOTE; ANIMAL; GENE EDITING; METABOLISM; MOUSE;

ADENINE; ANIMALS; GENE EDITING; MICE; ZYGOTE;

EID: 85051130181     PISSN: 1674800X     EISSN: 16748018     Source Type: Journal    
DOI: 10.1007/s13238-018-0566-z     Document Type: Letter

References (15)

1. Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S et al (2018) Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol 17(3):251–267
2. Faustino NA, Cooper TA (2003) Pre-mRNA splicing and human disease. Genes Dev 17:419–437
3. Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI, Liu DR (2017) Programmable base editing of A*T to G*C in genomic DNA without DNA cleavage. Nature 551:464–471
4. Hu JH, Miller SM, Geurts MH, Tang W, Chen L, Sun N, Zeina CM, Gao X, Rees HA, Lin Z et al (2018) Evolved Cas9 variants with broad PAM compatibility and high DNA specificity. Nature 556:57–63
5. Kim D, Kim S, Kim S, Park J, Kim JS (2016) Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq. Genome Res 26:406–415
6. Kim K, Ryu SM, Kim ST, Baek G, Kim D, Lim K, Chung E, Kim S, Kim JS (2017a) Highly efficient RNA-guided base editing in mouse embryos. Nat Biotechnol 35:435–437
7. Kim D, Lim K, Kim ST, Yoon SH, Kim K, Ryu SM, Kim JS (2017b) Genome-wide target specificities of CRISPR RNA-guided programmable deaminases. Nat Biotechnol 35:475–480
8. Komor AC, Kim YB, Packer MS, Zuris JA, Liu DR (2016) Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533:420–424
9. Komor AC, Badran AH, Liu DR (2017) CRISPR-Based technologies for the manipulation of eukaryotic genomes. Cell 169:559
10. Liang P, Zhang X, Chen Y, Huang J (2017a) Developmental history and application of CRISPR in human disease. J Gene Med. 10.1002/jgm.296
11. Liang P, Sun H, Sun Y, Zhang X, Xie X, Zhang J, Zhang Z, Chen Y, Ding C, Xiong Y et al (2017b) Effective gene editing by high-fidelity base editor 2 in mouse zygotes. Protein Cell 8:601–611
12. Liang P, Ding C, Sun H, Xie X, Xu Y, Zhang X, Sun Y, Xiong Y, Ma W, Liu Y et al (2017c) Correction of beta-thalassemia mutant by base editor in human embryos. Protein Cell 8:811–822
13. Liu Z, Lu Z, Yang G, Huang S, Li G, Feng S, Liu Y, Li J, Yu W, Zhang Y et al (2018) Efficient generation of mouse models of human diseases via ABE- and BE-mediated base editing. Nat Commun 9:2338
14. Ryu SM, Koo T, Kim K, Lim K, Baek G, Kim ST, Kim HS, Kim D, Lee H, Chung E et al (2018) Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy. Nat Biotechnol 36:536–539
15. Zhang X, Liang P, Ding C, Zhang Z, Zhou J, Xie X, Huang R, Sun Y, Sun H, Zhang J et al (2016) Efficient production of gene-modified mice using Staphylococcus aureus Cas9. Sci Rep 6:32565