基因编辑技术最新研究进展
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摘要
基因编辑是一种对基因组及其转录产物进行定点修饰、定向敲除或插入目的基因的基因编辑技术。近年来,基因编辑技术发展日新月异,不仅极大的推动了基因功能研究进程,同时为人类遗传疾病的治疗带来了曙光。综述了CRISPR/Cas9、CRISPR/Cpf1、Ago/g DNA和SGN等技术的作用原理、优缺点、应用等,以期为相关领域的研究提供参考。
Gene editing is a powerful set of techniques for site-directed modification and knockout or insertion of the genes on genomeor its transcripts. Recently,gene editing techniques have been greatly developed,which not only significantly promotes the process of genefunction investigation,but also shows the potential applications for human genetic disease treatments. Here,we summarized the principles,advantages,disadvantages,and applications of gene editing methods including CRISPR/Cas9,CRISPR/Cpf1,Ago/g DNA and SGN in orderto provide necessary informations on gene editing for the relevant scientific research.
Gene editing is a powerful set of techniques for site-directed modification and knockout or insertion of the genes on genomeor its transcripts. Recently,gene editing techniques have been greatly developed,which not only significantly promotes the process of genefunction investigation,but also shows the potential applications for human genetic disease treatments. Here,we summarized the principles,advantages,disadvantages,and applications of gene editing methods including CRISPR/Cas9,CRISPR/Cpf1,Ago/g DNA and SGN in orderto provide necessary informations on gene editing for the relevant scientific research.
引文
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[26]Gao F,Shen XZ,Jiang F,et al.DNA-guided genome editing usingthe Natronobacterium gregoryi Argonaute[J].Nat Biotechnol,2016,34(7):768-773.
[27]Qi J,Dong Z,Shi Y,et al.Ng Ago-based fabp11a gene knockdowncauses eye developmental defects in zebrafish[J].Cell Res,2016,26(12):1349-1352.
[28]Burgess S,Cheng L,Gu F,et al.Questions about Ng Ago[J].Protein Cell,2016,7(12):913-915.
[29]Lee SH,Turchiano G,Ata H,et al.Failure to detect DNA-guidedgenome editing using Natronobacterium gregoryi Argonaute[J].Nat Biotechnol,2016,35(1):17-18.
[30]Imanishi M,Nakamura A,Morisaki T,et al.Positive and negativecooperativity of modularly assembled zinc fingers[J].BiochemBioph Res Co,2009,387(3):440-443.
[31]Maeder ML,Thibodeau-Beganny S,Osiak A,et al.Rapid“opensource”engineering of customized zinc-finger nucleases for highlyefficient gene modification[J].Mol Cell,2008,31(2):294-301.
[32]Juillerat A,Dubois G,Valton J,et al.Comprehensive analysis of thespecificity of transcription activator-like effector nucleases[J].Nucleic Acids Res,2014,42(8):5390-5402.
[33]Smith J,Grizot S,Arnould S,et al.A combinatorial approachto create artificial homing endonucleases cleaving chosensequences[J].Nucleic Acids Res,2006,34(22):e149.
[34]Harrington JJ,Lieber MR.The characterization of a mammalianDNA structure-specific endonuclease[J].EMBO J,1994,13(5):1235-1246.
[35]Kaiser MW,Lyamicheva N,Ma W,et al.A comparison ofeubacterial and archaeal structure-specific 5'-exonucleases[J].JBiol Chem,1999,274(30):21387-21394.
[36]Kao HI,Henricksen LA,Liu Y,et al.Cleavage specificity ofSaccharomyces cerevisiae flap endonuclease 1 suggests a doubleflap structure as the cellular substrate[J].J Biol Chem,2002,277(17):14379-14389.
[37]Xu S,Cao S,Zou B,et al.An alternative novel tool for DNAediting without target sequence limitation:the structure-guidednuclease[J].Genome Biol,2016,17(1):186.
[38]Isabella Wolcott.Could the CRISPR-Cas9 genome editing systembe replaced by the Ng Ago-g DNA system?[J].Express Biology,2016,1(1):10-15.
[2]Capecchi MR.Altering the genome by homologous recombination[J].Science,1989,244(4910):1288-1292.
[3]Wood AJ,Lo TW,Zeitler B,et al.Targeted genome editing acrossspecies using ZFNs and TALENs[J].Science,2011,333(6040):307-307.
[4]Lo TW,Pickle CS,Lin S,et al.Precise and heritable genome editingin evolutionarily diverse nematodes using TALENs and CRISPR/Cas9 to engineer insertions and deletions[J].Genetics,2013,195(2):331-348.
[5]Urnov FD,Miller JC,Lee YL,et al.Highly efficient endogenoushuman gene correction using designed zinc-finger nucleases[J].Nature,2005,435(7042):646-651.
[6]Miller JC,Holmes MC,Wang J,et al.An improved zinc-fingernuclease architecture for highly specific genome editing[J].NatBiotechnol,2007,25(7):778-785.
[7]Boch J,Scholze H,Schornack S,et al.Breaking the code of DNAbinding specificity of TAL-typeⅢeffectors[J].Science,2009,326(5959):1509-1512.
[8]Jinek M,Chylinski K,Fonfara I,et al.A programmable dual-RNAguided DNA endonuclease in adaptive bacterial immunity[J].Science,2012,337(6069):816-821.
[9]Hsu PD,Lander ES,Zhang F.Development and applications ofcrispr-cas9 for genome engineering[J].Cell,2014,157(6):1262-1278.
[10]Chylinski K,Le Rhun A,Charpentier E.The tracr RNA and Cas9families of typeⅡCRISPR/Cas immunity systems[J].RNABiol,2013,10(5):726-737.
[11]Niu Y,Shen B,Cui Y,et al.Generation of gene-modifiedcynomolgus monkey via Cas9/RNA-mediated gene targeting in onecell embryos[J].Cell,2014,156(4):836-843.
[12]Liang P,Xu Y,Zhang X,et al.CRISPR/Cas9-mediated gene editingin human tripronuclear zygotes[J].Protein Cell,2015,6(5):363-372.
[13]Kang X,He W,Huang Y,et al.Introducing precise geneticmodifications into human 3PN embryos by CRISPR/Cas-mediatedgenome editing[J].J Assist Reprod Gen,2016,33(5):581-588.
[14]Zhu S,Li W,Liu J,et al.Genome-scale deletion screening ofhuman long non-coding RNAs using a paired-guide RNA CRISPRCas9 library[J].Nat Biotechnol,2016,34(12):1279-1286.
[15]Cyranoski D.CRISPR gene-editing tested in a person for the firsttime[J].Nature,2016,539(7630):479.
[16]Zetsche B,Gootenberg JS,Abudayyeh OO,et al.Cpf1 is a singleRNA-guided endonuclease of a class 2 CRISPR-Cas system[J].Cell,2015,163(3):759-771.
[17]Dong D,Ren K,Qiu X,et al.The crystal structure of Cpf1 incomplex with CRISPR RNA[J].Nature,2016,532(7600):522-526.
[18]Mali P,Aach J,Stranges PB,et al.CAS9 transcriptional activatorsfor target specificity screening and paried nickases for cooperativegenome engineering[J].Nat Biotechnol,2013,31(9):833-838.
[19]Kim D,Kim J,Hur JK,et al.Genome-wide analysis revealsspecificities of Cpf1 endonucleases in human cells[J].NatBiotechnol,2016,34(8):863-868.
[20]Song JJ,Smith KK,Hannon GJ,et al.Crystal structure of Argonauteand its implications for RISC slicer activity[J].Science,2004,305(5689):1434-1437.
[21]Yuan YR,Pei Y,Ma JB,et al.Crystal structure of A.aeolicusargonaute,a site-specific DNA-guided endoribonuclease,providesinsights into RISC-mediated MRNA cleavage[J].Mol Cell,2005,19(3):405-419.
[22]Junho KH,Ivan O,Alexei AA.Prokaryotic Argonautes defendgenomes against invasive DNA[J].Trend Biochem Sci,2014,39(6):257-259.
[23]Meister G.Argonaute protein:Functinal insights and emergingroles[J].Nat Rev Genet,2013,14(7):447-459.
[24]Swarts DC,Jore MM,Westra ER,et al.DNA-guided DNAinterference by a prokaryotic Argonaute[J].Nature,2014,507(7491):258-261.
[25]Daan CS,Jorrit WH,Ismael H,et al.Argonaute of the archaeonPyrococcus furiosus is a DNA-guided nuclease that targets cognateDNA[J].Nucleic Acids Res,2015,43(10):5120-5129.
[26]Gao F,Shen XZ,Jiang F,et al.DNA-guided genome editing usingthe Natronobacterium gregoryi Argonaute[J].Nat Biotechnol,2016,34(7):768-773.
[27]Qi J,Dong Z,Shi Y,et al.Ng Ago-based fabp11a gene knockdowncauses eye developmental defects in zebrafish[J].Cell Res,2016,26(12):1349-1352.
[28]Burgess S,Cheng L,Gu F,et al.Questions about Ng Ago[J].Protein Cell,2016,7(12):913-915.
[29]Lee SH,Turchiano G,Ata H,et al.Failure to detect DNA-guidedgenome editing using Natronobacterium gregoryi Argonaute[J].Nat Biotechnol,2016,35(1):17-18.
[30]Imanishi M,Nakamura A,Morisaki T,et al.Positive and negativecooperativity of modularly assembled zinc fingers[J].BiochemBioph Res Co,2009,387(3):440-443.
[31]Maeder ML,Thibodeau-Beganny S,Osiak A,et al.Rapid“opensource”engineering of customized zinc-finger nucleases for highlyefficient gene modification[J].Mol Cell,2008,31(2):294-301.
[32]Juillerat A,Dubois G,Valton J,et al.Comprehensive analysis of thespecificity of transcription activator-like effector nucleases[J].Nucleic Acids Res,2014,42(8):5390-5402.
[33]Smith J,Grizot S,Arnould S,et al.A combinatorial approachto create artificial homing endonucleases cleaving chosensequences[J].Nucleic Acids Res,2006,34(22):e149.
[34]Harrington JJ,Lieber MR.The characterization of a mammalianDNA structure-specific endonuclease[J].EMBO J,1994,13(5):1235-1246.
[35]Kaiser MW,Lyamicheva N,Ma W,et al.A comparison ofeubacterial and archaeal structure-specific 5'-exonucleases[J].JBiol Chem,1999,274(30):21387-21394.
[36]Kao HI,Henricksen LA,Liu Y,et al.Cleavage specificity ofSaccharomyces cerevisiae flap endonuclease 1 suggests a doubleflap structure as the cellular substrate[J].J Biol Chem,2002,277(17):14379-14389.
[37]Xu S,Cao S,Zou B,et al.An alternative novel tool for DNAediting without target sequence limitation:the structure-guidednuclease[J].Genome Biol,2016,17(1):186.
[38]Isabella Wolcott.Could the CRISPR-Cas9 genome editing systembe replaced by the Ng Ago-g DNA system?[J].Express Biology,2016,1(1):10-15.