动物基因组编辑及其应用研究进展
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摘要
基因组编辑技术已成为研究人员进行生物和生物医学研究的强大武器。为了进一步改善和拓展其功能,科学家们在多种生物个体上进行了测试,优化了多种基因编辑工具递送策略,并且已经研发出了多种方案来确保基因组编辑的准确性。本研究回顾了基因组编辑技术的历史里程碑,讨论了出现的相关技术应用,最后,本研究还预测了未来基因组编辑技术的发展方向,为基因组编辑技术的未来发展提供了一个崭新的视角。
Genome editing technology has become a powerful weapon for researchers in biological and biomedical research. In order to further improve and expand its functions, scientists have carried out tests on a variety of biological individuals, optimized the delivery strategies of various gene editing tools, and have developed a variety of schemes to ensure the accuracy of genome editing. This study reviewed the historic milestones of genome editing technology and discussed the related technical applications which have emerged.Finally, this research also predicted the future development direction of genome editing technology, and would provide a new perspective for the future development of genome editing technology.
Genome editing technology has become a powerful weapon for researchers in biological and biomedical research. In order to further improve and expand its functions, scientists have carried out tests on a variety of biological individuals, optimized the delivery strategies of various gene editing tools, and have developed a variety of schemes to ensure the accuracy of genome editing. This study reviewed the historic milestones of genome editing technology and discussed the related technical applications which have emerged.Finally, this research also predicted the future development direction of genome editing technology, and would provide a new perspective for the future development of genome editing technology.
引文
Bassett A.R.,Tibbit C.,Ponting C.P.,and Liu J.L.,2013,Highly efficient targeted mutagenesis of Drosophila with the CRISP-R/Cas9 system,Cell Rep.,4(1):220-228
Bi Y.Z.,Hua Z.D.,Liu X.M.,Hua W.J.,Ren H.Y.,Xiao H.W.,Zhang L.P.,Li L.,Wang Z.R.,Laible G.,Wang Y.,Dong F.,and Zheng X.M.,2016,Isozygous and selectable marker-free MSTN knockout cloned pigs generated by the com bined use of CRISPR/Cas9 and Cre/LoxP,Sci.Rep.,6:31729
Carroll D.,2011,Genome engineering with zinc-finger nucleases,Genetics,188(4):773-782
Chandrasegaran S.,and Carroll D.,2016,Origins of programmable nucleases for genome engineering,J.Mol.Biol.,428(5 Pt B):963-989
Clark J.,and Whitelaw B.,2003,A future for transgenic livestock,Nat.Rev.Genet.,4(10):825-833
Di Carlo J.E.,Norville J.E.,Mali P.,Rios X.,Aach J.,and Church G.M.,2013,G enome engineering in saccharomyces cerevisiae using CRISPR-Cas systems,Nucleic Acids Res.,41(7):4336-4343
Fineran P.C.,and Charpentier E.,2012,Memory of viral infections by CRISPR-Cas adaptive immune systems:acquisition of new information,Virology,434(2):202-209
Gaj T.,Gersbach C.A.,and Lii C.F.B.,2013,ZFN,TALEN,and CRISPR/Cas-based methods for genome engineering,Trends Biotechnol.,31(7):397-405
Hai T.,Teng F.,Guo R.,Li W.,and Zhou Q.,2014,One-step generation of knockout pigs by zygote injection of CRISPR/Cas system,Cell Res.,24(3):372-375
Hauschild I.,Vitzthum K.,Klapp B.F.,Groneberg D.A.,and Mache S.,2011,Time and motion study of anesthesiologists'workflow in German hospitals,Wien.Med.Wochenschr.,161(17-18):433-440
Hsu P.D.,Scott D.A.,Weinstein J.A.,Ran F.A,Konermann S.,Agarwala V.,Li Y.Q.,Fine E.J.,Wu X.B.,Shalem O.,Cradick T.J.,Marraffini L.A.,Bao G.,and Zhang F.,2013,DNA targeting specificity of RNA-guided Cas9 nucleases,Nat.Biotechnol.,31(9):827-832
Hwang W.Y.,Fu Y.F.,Reyon D.,Maeder M.L.,Tsai S.Q.,Sander J.D.,Peterson R.T.,Yeh J.R.J.and Joung J.K.,2013,Efficient genome editing in zebrafish using a CRISPR-Cas system,Nat.Biotechnol.,31(3):227
Jiang W.Y.,Bikard D.,Cox D.,Zhang F.,and Marraffini L.A.,2013,RNA-guided editing of bacterial genomes using CRISPR-Cas systems,Nat.Biotechnol.,31(3):233-239
Kim H.,and Kim J.S.,2014,A guide to genome engineering with programmable nucleases,Nat.Rev.Genet.,15(5):321-334
Li D.,Qiu Z.,Shao Y.,Chen Y.,Guan Y.,Liu M.,Li Y.,Gao N.,Wng L.,Lu X.,Zhao Y.,and Liu M.,2013b,Heritable gene targeting in the mouse and rat using a CRISPR-Cas system,Nat.Biotechnol.,31(8):681-683
Li J.F.,Aach J.,Norville J.E..,McCormack M.,Zhang D.D.,Bush J.,Church G.M.,and Sheen J.,2013a,Multiplex and homologous recombination-mediated plant genome editing in Arabidopsis and nicotiana benthamiana using guide RNAand Cas9,Nat.Biotechnol.,31(8):688-691
Li T.,Huang S.,Jiang W.Z.,Wright D.,Spalding M.H.,Weeks D.P.,and Yang B.,2010,TAL nucleases(TALNs):hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain,Nucleic Acids Res.,39(1):359-372
Lillico S.G.,Proudfoot C.,Carlson D.F.,Stverakova D.,Neil C.,Blain C.,King T.J.,Ritchie W.A.,Tan W.F.,Mileham A.J.,McLaren D.G.,Fahrenkrug S.C.,and Whitelaw C.B.A.,2013,Live pigs produced from genome edited zygotes,Sci.Rep.,3(7470):2847
Lin Y.N.,Cradick T.J.,Brown M.T.,Deshmukh H.,Ranjan P.,Sarode N.,Wile B.M.,Vertino P.M.,Stewart F.J.,and Bao G.,2014,CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences,Nucleic Acids Res.,42(11):7473-7485
Ma Y.W.,Shen B.,Zhang X.,Lu Y.D.,Chen W.,Ma J.,Huang X.X.,and Zhang L.F.,2014,Heritable multiplex genetic engineering in rats using CRISPR/Cas9,PLoS One,9(3):e89413
Mali P.,Yang L.H.,Esvelt K.M.,Aach J.,Guell M.,Di Carlo J.E.,Norville J.E.,and Church G.M.,2013,RNA-guided human genome engineering via Cas9,Science,339(6121):823
Niu Y.,Shen B.,Cui Y.,Chen Y.,Wang J.,Wang L.,Kang Y.,Zhao X.,Si W.,Li W.,Xiang A.P.,Zhou J.,Guo X.,Bi Y.,Si C.,Hu B.,Dong G.,Wang H.,Zhou Z.,Li T.,Tan T.,Pu X.,Wang F.,Ji S.,Zhou Q.,Huang X.,Ji W.,and Sha J.,2014,Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos,Cell,156(4):836-843
Peng J.,Wang Y.,Jiang J.,Zhou X.,Song L.,Wang L.,Ding C.,Qin J.,Liu L.,Wang W.,Liu J.,Huang X.,Wei H.,and Zhang P.,2015,Producti on of human albumin in pigs through CRISPR/Cas9-mediated knockin of human cDNAinto swine albumin locus in the zygotes,Sci.Rep.,5:16705
Shen B.,Zhang J.,Wu H.,Wang J.,Ma K.,Li Z.,Zhang X.,Zhang P.,and Huang X.,2013,Generation of gene-modified mice via Cas9/RNA-mediated gene targeting,Cell Res.,23(5):720-723
Tebas P.,Stein D.,Tang W.W.,Frank I.,Wang S.Q.,Lee G.,Spratt S.K.,Surosky R.T.,Giedlin M.A.,Nichol G.,Holmes M.C.,Gregory P.D.,Ando D.G.,Kalos M.,Collman R.G.,Binder-Scholl G.,Plesa G.,Hwang W.T.,Levine B.L.,and June C.H.,2014,Gene editing of CCR5 in autologous CD4T cells of persons infected with HIV,N.Engl.J.Med.,370(10):901-910
Urnov F.D.,Miller J.C.,Lee Y.L.,Beausejour C.M.,Rock J.M.,Augustus S.,Jamieson A.C.,Porteus M.H.,Gregory P.D.,and Holmes M.C.,2005a,Highly efficient endogenous human gene cor rection using designed zinc-finger nucleases,Nature,435(7042):646-651
Urnov F.D.,Rebar E.J.,Holmes M.C.,Zhang H.S.,and Gregory P.D.,2010b,Genome editing with engineered zinc finger nucleases,Nat.Rev.Genet.,11(9):636-646
Xiao A.,Wang Z.X.,Hu Y.Y.,Wu Y.D.,Luo Z.,Yang Z.P.,Zu Y.,Li W.Y.,Huang P.,Tong X.J.,Zhu Z.Y.,Lin S.,and Zhang B.,2013,Chromosomal deletions and inversions mediated by TALENs and CRISPR/Cas in zebrafish,Nucleic Acids Res.,41(14):e141
Yang D.,Xu J.,Zhu T.,Fan J.,Lai L.,Zhang J.,and Chen Y.E.,2014,Effective gene targeting in rabbits using RNA-guided Cas9 nucleases,J.Mol.Cell Biol.,6(1):97-99
Zhou J.,Shen B.,Zhang W.,Wang J.,Yang J.,Chen L.,Zhang N.,Zhu K.,Xu J.,Hu B.,Leng Q.,and Huang X.,2013,One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering,Int.J.Biochem.Cell Biol.,46(1):49-55
Bi Y.Z.,Hua Z.D.,Liu X.M.,Hua W.J.,Ren H.Y.,Xiao H.W.,Zhang L.P.,Li L.,Wang Z.R.,Laible G.,Wang Y.,Dong F.,and Zheng X.M.,2016,Isozygous and selectable marker-free MSTN knockout cloned pigs generated by the com bined use of CRISPR/Cas9 and Cre/LoxP,Sci.Rep.,6:31729
Carroll D.,2011,Genome engineering with zinc-finger nucleases,Genetics,188(4):773-782
Chandrasegaran S.,and Carroll D.,2016,Origins of programmable nucleases for genome engineering,J.Mol.Biol.,428(5 Pt B):963-989
Clark J.,and Whitelaw B.,2003,A future for transgenic livestock,Nat.Rev.Genet.,4(10):825-833
Di Carlo J.E.,Norville J.E.,Mali P.,Rios X.,Aach J.,and Church G.M.,2013,G enome engineering in saccharomyces cerevisiae using CRISPR-Cas systems,Nucleic Acids Res.,41(7):4336-4343
Fineran P.C.,and Charpentier E.,2012,Memory of viral infections by CRISPR-Cas adaptive immune systems:acquisition of new information,Virology,434(2):202-209
Gaj T.,Gersbach C.A.,and Lii C.F.B.,2013,ZFN,TALEN,and CRISPR/Cas-based methods for genome engineering,Trends Biotechnol.,31(7):397-405
Hai T.,Teng F.,Guo R.,Li W.,and Zhou Q.,2014,One-step generation of knockout pigs by zygote injection of CRISPR/Cas system,Cell Res.,24(3):372-375
Hauschild I.,Vitzthum K.,Klapp B.F.,Groneberg D.A.,and Mache S.,2011,Time and motion study of anesthesiologists'workflow in German hospitals,Wien.Med.Wochenschr.,161(17-18):433-440
Hsu P.D.,Scott D.A.,Weinstein J.A.,Ran F.A,Konermann S.,Agarwala V.,Li Y.Q.,Fine E.J.,Wu X.B.,Shalem O.,Cradick T.J.,Marraffini L.A.,Bao G.,and Zhang F.,2013,DNA targeting specificity of RNA-guided Cas9 nucleases,Nat.Biotechnol.,31(9):827-832
Hwang W.Y.,Fu Y.F.,Reyon D.,Maeder M.L.,Tsai S.Q.,Sander J.D.,Peterson R.T.,Yeh J.R.J.and Joung J.K.,2013,Efficient genome editing in zebrafish using a CRISPR-Cas system,Nat.Biotechnol.,31(3):227
Jiang W.Y.,Bikard D.,Cox D.,Zhang F.,and Marraffini L.A.,2013,RNA-guided editing of bacterial genomes using CRISPR-Cas systems,Nat.Biotechnol.,31(3):233-239
Kim H.,and Kim J.S.,2014,A guide to genome engineering with programmable nucleases,Nat.Rev.Genet.,15(5):321-334
Li D.,Qiu Z.,Shao Y.,Chen Y.,Guan Y.,Liu M.,Li Y.,Gao N.,Wng L.,Lu X.,Zhao Y.,and Liu M.,2013b,Heritable gene targeting in the mouse and rat using a CRISPR-Cas system,Nat.Biotechnol.,31(8):681-683
Li J.F.,Aach J.,Norville J.E..,McCormack M.,Zhang D.D.,Bush J.,Church G.M.,and Sheen J.,2013a,Multiplex and homologous recombination-mediated plant genome editing in Arabidopsis and nicotiana benthamiana using guide RNAand Cas9,Nat.Biotechnol.,31(8):688-691
Li T.,Huang S.,Jiang W.Z.,Wright D.,Spalding M.H.,Weeks D.P.,and Yang B.,2010,TAL nucleases(TALNs):hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain,Nucleic Acids Res.,39(1):359-372
Lillico S.G.,Proudfoot C.,Carlson D.F.,Stverakova D.,Neil C.,Blain C.,King T.J.,Ritchie W.A.,Tan W.F.,Mileham A.J.,McLaren D.G.,Fahrenkrug S.C.,and Whitelaw C.B.A.,2013,Live pigs produced from genome edited zygotes,Sci.Rep.,3(7470):2847
Lin Y.N.,Cradick T.J.,Brown M.T.,Deshmukh H.,Ranjan P.,Sarode N.,Wile B.M.,Vertino P.M.,Stewart F.J.,and Bao G.,2014,CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences,Nucleic Acids Res.,42(11):7473-7485
Ma Y.W.,Shen B.,Zhang X.,Lu Y.D.,Chen W.,Ma J.,Huang X.X.,and Zhang L.F.,2014,Heritable multiplex genetic engineering in rats using CRISPR/Cas9,PLoS One,9(3):e89413
Mali P.,Yang L.H.,Esvelt K.M.,Aach J.,Guell M.,Di Carlo J.E.,Norville J.E.,and Church G.M.,2013,RNA-guided human genome engineering via Cas9,Science,339(6121):823
Niu Y.,Shen B.,Cui Y.,Chen Y.,Wang J.,Wang L.,Kang Y.,Zhao X.,Si W.,Li W.,Xiang A.P.,Zhou J.,Guo X.,Bi Y.,Si C.,Hu B.,Dong G.,Wang H.,Zhou Z.,Li T.,Tan T.,Pu X.,Wang F.,Ji S.,Zhou Q.,Huang X.,Ji W.,and Sha J.,2014,Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos,Cell,156(4):836-843
Peng J.,Wang Y.,Jiang J.,Zhou X.,Song L.,Wang L.,Ding C.,Qin J.,Liu L.,Wang W.,Liu J.,Huang X.,Wei H.,and Zhang P.,2015,Producti on of human albumin in pigs through CRISPR/Cas9-mediated knockin of human cDNAinto swine albumin locus in the zygotes,Sci.Rep.,5:16705
Shen B.,Zhang J.,Wu H.,Wang J.,Ma K.,Li Z.,Zhang X.,Zhang P.,and Huang X.,2013,Generation of gene-modified mice via Cas9/RNA-mediated gene targeting,Cell Res.,23(5):720-723
Tebas P.,Stein D.,Tang W.W.,Frank I.,Wang S.Q.,Lee G.,Spratt S.K.,Surosky R.T.,Giedlin M.A.,Nichol G.,Holmes M.C.,Gregory P.D.,Ando D.G.,Kalos M.,Collman R.G.,Binder-Scholl G.,Plesa G.,Hwang W.T.,Levine B.L.,and June C.H.,2014,Gene editing of CCR5 in autologous CD4T cells of persons infected with HIV,N.Engl.J.Med.,370(10):901-910
Urnov F.D.,Miller J.C.,Lee Y.L.,Beausejour C.M.,Rock J.M.,Augustus S.,Jamieson A.C.,Porteus M.H.,Gregory P.D.,and Holmes M.C.,2005a,Highly efficient endogenous human gene cor rection using designed zinc-finger nucleases,Nature,435(7042):646-651
Urnov F.D.,Rebar E.J.,Holmes M.C.,Zhang H.S.,and Gregory P.D.,2010b,Genome editing with engineered zinc finger nucleases,Nat.Rev.Genet.,11(9):636-646
Xiao A.,Wang Z.X.,Hu Y.Y.,Wu Y.D.,Luo Z.,Yang Z.P.,Zu Y.,Li W.Y.,Huang P.,Tong X.J.,Zhu Z.Y.,Lin S.,and Zhang B.,2013,Chromosomal deletions and inversions mediated by TALENs and CRISPR/Cas in zebrafish,Nucleic Acids Res.,41(14):e141
Yang D.,Xu J.,Zhu T.,Fan J.,Lai L.,Zhang J.,and Chen Y.E.,2014,Effective gene targeting in rabbits using RNA-guided Cas9 nucleases,J.Mol.Cell Biol.,6(1):97-99
Zhou J.,Shen B.,Zhang W.,Wang J.,Yang J.,Chen L.,Zhang N.,Zhu K.,Xu J.,Hu B.,Leng Q.,and Huang X.,2013,One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering,Int.J.Biochem.Cell Biol.,46(1):49-55
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