基因编辑技术及其应用的研究进展

doi:10.16431/j.cnki.1671-7236.2017.08.004

Abstract

Abstract:

Gene editing techniques (GETs) can introduce mutations precisely into endogenous genome at a designed location. GETs are widely used in the research of bio-sciences, and promote a great development in life sciences. So far, most of the artificial gene mutations are generated by the following three GETs, including zinc finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeats/Cas endonuclease (CRISPR/Cas). GETs can recognize and modify the target sequence of endogenous gene with specificity, which are good ways to study the function of new genes. GETs have the advantage of simple operation, high efficiency and accuracy, so it is widely used in life science research, constraction of disease models, drug research and development and agricultural production. This review presents the three different nuclease-based GETs,including the mechanisms by which they produce genetic modifications, their recent advances and prospects as well as the differences between GETs and transgene techniques.

Key words: gene editing; ZFN; TALEN; CRISPR/Cas

CLC Number:

LI Xiang, CUI Wen-tao, LI Kui. Research Advances on Techniques of Gene Editing and Its Application[J]. , 2017, 44(8): 2241-2247.

References

[1] Thomas K R,Folger K R,Capecchi M R. High frequency targeting of genes to specific sites in the mammalian genome[J].Cell,1986, 44(3):419-428.
[2] Gao F, Shen X, Jiang F, et al. DNA-guided genome editing using the Natronobacterium gregoryi Argonaute[J].Nature Biotechnology, 2016,34:768-773.
[3] Miller J, McLaehlan A D, Klug A, et al.Repetitive zinc binding domains in the protein transcription factor Ⅲ A from Xenopus oocytes[J].EMBO J,1985,4(6):1609-1614.
[4] Klug A. The discovery of zine fingers and their development for practical applications in gene regulation and genome manipulation[J].Q Rev Biophys, 2010,43(1):1-21.
[5] Wolfe S A, Neklndov A L, Pabo C O, et al. DNA recognition by Cys2/His2 zinc finger protein[J].Annu Rev Biophys Biomol Struct, 2000,29(6):183-212.
[6] Joel P, David J. Engineered Zinc Finger Proteins:Methods and Protocols[M]. Germany:Humana Press,2010.
[7] 刘蓓,尉玮,王丽华.基因编辑新技术研究进展[J]. 亚热带农业研究,2013,9(4):262-268.
[8] 肖安, 胡莹莹,林硕,等.人工锌指核酸酶介导的基因组定点修饰技术[J].遗传,2011,33(7):665-683.
[9] Ramirez C L, Foley J E, Joung J K, et al. Unexpected failure rates for modular assembly of engineered zinc fingers[J].Nature Methods,2008,5(5):374-375.
[10] Maeder M L, Thibodean-Beganny S, Joung J K, et al. Rapid "Open-Source" engineering of customized zine-finger nucleases for highly efficient gene modification[J].Mol Cell, 2008, 31(2):294-301.
[11] Meng X D, Noyes M B, Zhu L J, et al. Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases[J].Nature Biontechnology,2008,26(6):695-701.
[12] Sander J D, Dahlborg E J, Joung J K, et al. Selection-free zinc-finger-nuclease engineering context-dependent assembly(CoDA)[J]. Nature Methods,2011,8(1):67-69.
[13] Vanamee E S, Santagata S, Aggarwal A K. Fok Ⅰ requires two specific DNA sites for cleavage[J]. J Mol Biol, 2001,309(1):69-78.
[14] Moscou M J, Bogdanove A J. A simple cipher governs DNA recognition by TAL effectors[J]. Science, 2009,326(5959):1501.
[15] Boch J, Scholze H, Schornack S, et al. Breaking the codeof DNA binding specificity of TAL-type Ⅲ effectors[J].Science, 2009, 326(5959):1509-1512.
[16] Christian M, Cermak T, Doyle E L, et al. Targeting DNA double-strand breaks with TAL effector nucleases[J].Genetics, 2010, 186(2):757-761.
[17] Bonas U, Stall R E, Staskawicz B. Genetic and structural characterization of the avirulence gene AvrBs3 from Xanthomonas campestris pv. vesicatoria[J].Mol Gen Genet,1989,218(1):127-136.
[18] Boch J, Bonas U. Xanthomonas AvrBs3 family-type Ⅲ effectors:Discovery and function[J]. Annu Rev Phytopathol,2010,48:419-436.
[19] Glazkova D V, Shipulin G A.TALE nucleases as a new tool for genome editing[J]. Molecular Biology,2014,48(3):355-370.
[20] Cermak T, Doyle E L, Christian M, et al. Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting[J].Nucleic Acids Res,2011,39(12):82.
[21] Sander J D, Cade L, Khayter C, et al. Targeted gene disruption in somatic zebrafish cells using engineered TALENs[J]. Nat Biotechnol,2011,29(8):697-698.
[22] Reyon D, Tsai S Q, Khayter C, et al. FLASH assembly of TALENs for high through put genome editing[J].Nat Biotechnol,2012,30(5):460-465.
[23] Schmid-Burgk J L, Schmidt T, Kaiser V, et al. A ligation-independent cloning technique for high through put assembly of transcription activator-like effector genes[J]. Nat Biotechnol,2013, 31(1):76-81.
[24] Ishino Y,Shinagawa H, Makino K. Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli and identification of the gene product[J]. JBacteriol, 1987, 169(12):5429-5433.
[25] Mojica F J, Diez V C, Garcia M J. Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements[J]. J Mol Evol,2005, 60(2):174-182.
[26] Jansen R, Embden J D, Gaastra W, et al. Identification of genes that are associated with DNA repeats in prokaryotes[J].Mol Microbiol,2002,43(6):1565-1575.
[27] Deltcheva E, Chylinski K, Sharma C M. CRISPRRNA maturation by trans-encoded small RNA andhost factor RNase Ⅲ[J]. Nature, 2011, 471(7340):602-607.
[28] Chen-Tsai R Y, Jiang R, Zhuang L, et al. Genome editing and animal models[J].Chinese Bulletin, 2014, 59(1):1-6.
[29] Villion M, Moineau S. The double-edged sword of CRISPR-Cas systems[J]. Cell Res, 2013, 23(1):15-17.
[30] Jinek M, Chylinski K, Fonfara I. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity[J]. Science, 2012, 337(6096):816-821.
[31] Waltz E. Gene-edited CRISPR mushroom escapes US regulation(N)[J].Nature,2016,532(7599):293.
[32] Huang S, Weigel D, Beachy R N, et al. A proposed regulatory framework for genome-edited crops[J]. Nature Genetics,2016,48(2):109-111.
[33] Perez E E, Wang J, Miller J C, et al.Establishment of HIV-1 resistance in CD4⁺ T cells by genome editing using zinc-finger nucleases[J].Nat Biotechnol,2008,26(7):808-816.
[34] Bacman S R, Williams S L, Pinto M, et al. Specific elimination of mutant mitochondrial genomes in patient-derived cells by mito TALENs[J]. Nat Med,2013,19(9):1111-1113.
[35] Wu Y, Liang D, Wang Y,et al. Correction of a genetic disease in mouse via use of CRISPR-Cas9[J]. Cell Stem Cell, 2013, 13(6):659-662.
[36] Long C, McAnally J R, Shelton J M, et al. Prevention of muscular dystrophy in mice by CRSIPR/Cas9-mediated editing of germline DNA[J].Science,2014,345(6201):1184-1188.
[37] 梁普平,黄军就.推开人类胚胎基因研究的神秘大门[J].中国生命科学通报,2016,28(4):421-425.
[38] Townsend J A, Wright D A, Winfrey R J, et al.High-frequency modification of plant genes using engineered zinc-finger nucleases[J]. Nature, 2009,459(7245):442-445.
[39] Li T, Liu B, Spalding M H, et al. High-efficiency TALEN-based gene editing produces disease-resistant rice[J]. Nature Biotechnology,2012, 30(5):390-392.
[40] Liu X, Wang Y S, Tian Y C, et al. Generation of mastitis resistance in cows by targeting human lysozyme geneto beta-casein locus using zinc-finger nucleases[J].Proc Biol Sci, 2014,281(1780):20133368.
[41] Qian L L, Tang M X, Yang J Z,et al. Targeted mutations in myostatin by zinc-finger nucleases result in double-muscled phenotype in Meishan pigs[J].Scientific Reports,2015,5:14435.
[42] 李聪, 曹文广.CRISPR/Cas介导绵羊胎儿成纤维细胞肌肉生长抑制素基因敲除研究[J].中国畜牧兽医,2015,42(11):2813-2821.
[43] Tan W F, Carlson D F, Lanctoc C A, et al.Efficient nonmeiotic allele introgression in livestock using custom endonucleases[J].PNAS, 2013,110(41):16526-16531.
[44] Li W, Teng F, Tianda L,et al. Simultaneous generation and germline transmission of multiple gene mutations in rat using CRISPR-Cas systems[J].Nat Biotechnol,2013,31(8):684-686.
[45] Liu D Y, Qiu T D, Xiao H, et al. Rapid construction of multiple sgRNA vectors and high efficient knockout of Arabidopsis IAA2 gene using the CRISPR/Cas9 genomic editing technology[J].Hereditas,2016,38(8):756.
[46] Yang D S,Yang H Q, Wei L,et al.Generation of PPARgamma mono-allelic knockout pigs via zincfinger nucleases and nuclear transfer cloning[J]. Cell Res,2011,21(6):979-982.
[47] Park C Y, Kim J, Kweon J,et al. Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs[J].Proc Natl Acad Sci USA,2014,111(25):9253-9258.
[48] Platt R J,Chen S,Zhang F,et al.CRISPR-Cas9 knockin mice for genome editing and cancer modeling[J] Cell,2014,159(2):440-455.

Research Advances on Techniques of Gene Editing and Its Application

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