최소 단어 이상 선택하여야 합니다.
최대 10 단어까지만 선택 가능합니다.
다음과 같은 기능을 한번의 로그인으로 사용 할 수 있습니다.
NTIS 바로가기The Korean journal of internal medicine : KJIM, v.32 no.1, 2017년, pp.42 - 61
Kim, Eun Ji (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, Korea) , Kang, Ki Ho (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, Korea) , Ju, Ji Hyeon (Division of Rheumatology, Department of Internal Medicine, College of Medicine, Seoul St. Mary’s Hospital, The Catholic University of Korea, Seoul, Korea)
Recent advances in genome editing with programmable nucleases have opened up new avenues for multiple applications, from basic research to clinical therapy. The ease of use of the technology—and particularly clustered regularly interspaced short palindromic repeats (CRISPR)—will allow ...
1 Collins FS Varmus H A new initiative on precision medicine N Engl J Med 2015 372 793 795 25635347
2 Lander ES Cutting the Gordian helix: regulating genomic testing in the era of precision medicine N Engl J Med 2015 372 1185 1186 25689017
3 Jameson JL Longo DL Precision medicine: personalized, problematic, and promising N Engl J Med 2015 372 2229 2234 26014593
4 Barrangou R Fremaux C Deveau H CRISPR provides acquired resistance against viruses in prokaryotes Science 2007 315 1709 1712 17379808
5 Ishino Y Shinagawa H Makino K Amemura M Nakata A Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product J Bacteriol 1987 169 5429 5433 3316184
6 Bolotin A Quinquis B Sorokin A Ehrlich SD Clustered regularly interspaced short palindrome repeats (CRISPRs) have spacers of extrachromosomal origin Microbiology 2005 151 Pt 8 2551 2561 16079334
7 Mojica FJ Diez-Villasenor C Garcia-Martinez J Soria E Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements J Mol Evol 2005 60 174 182 15791728
8 Pourcel C Salvignol G Vergnaud G CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA, and provide additional tools for evolutionary studies Microbiology 2005 151 Pt 3 653 663 15758212
9 Marraffini LA Sontheimer EJ CRISPR interference limits horizontal gene transfer in staphylococci by targeting DNA Science 2008 322 1843 1845 19095942
10 Hale CR Zhao P Olson S RNA-guided RNA cleavage by a CRISPR RNA-Cas protein complex Cell 2009 139 945 956 19945378
11 Brouns SJ Jore MM Lundgren M Small CRISPR RNAs guide antiviral defense in prokaryotes Science 2008 321 960 964 18703739
12 Jinek M Chylinski K Fonfara I Hauer M Doudna JA Charpentier E A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity Science 2012 337 816 821 22745249
13 Cong L Ran FA Cox D Multiplex genome engineering using CRISPR/Cas systems Science 2013 339 819 823 23287718
14 Mali P Yang L Esvelt KM RNA-guided human genome engineering via Cas9 Science 2013 339 823 826 23287722
15 Mojica FJ Diez-Villasenor C Soria E Juez G Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria Mol Microbiol 2000 36 244 246 10760181
16 Jansen R Embden JD Gaastra W Schouls LM Identification of genes that are associated with DNA repeats in prokaryotes Mol Microbiol 2002 43 1565 1575 11952905
17 Makarova KS Aravind L Grishin NV Rogozin IB Koonin EV A DNA repair system specific for thermophilic Archaea and bacteria predicted by genomic context analysis Nucleic Acids Res 2002 30 482 496 11788711
18 Haft DH Selengut J Mongodin EF Nelson KE A guild of 45 CRISPR-associated (Cas) protein families and multiple CRISPR/Cas subtypes exist in prokaryotic genomes PLoS Comput Biol 2005 1 e60 16292354
19 Garneau JE Dupuis ME Villion M The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA Nature 2010 468 67 71 21048762
20 Deltcheva E Chylinski K Sharma CM CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III Nature 2011 471 602 607 21455174
21 Sapranauskas R Gasiunas G Fremaux C Barrangou R Horvath P Siksnys V The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli Nucleic Acids Res 2011 39 9275 9282 21813460
22 Jinek M East A Cheng A Lin S Ma E Doudna J RNAprogrammed genome editing in human cells Elife 2013 2 e00471 23386978
23 Hwang WY Fu Y Reyon D Efficient genome editing in zebrafish using a CRISPR-Cas system Nat Biotechnol 2013 31 227 229 23360964
24 Wang H Yang H Shivalila CS One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering Cell 2013 153 910 918 23643243
25 Bassett AR Tibbit C Ponting CP Liu JL Highly efficient targeted mutagenesis of Drosophila with the CRISPR/Cas9 system Cell Rep 2013 4 220 228 23827738
26 Friedland AE Tzur YB Esvelt KM Colaiacovo MP Church GM Calarco JA Heritable genome editing in C. elegans via a CRISPR-Cas9 system Nat Methods 2013 10 741 743 23817069
27 Li JF Norville JE Aach J Multiplex and homologous recombination-mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9 Nat Biotechnol 2013 31 688 691 23929339
28 Nakayama T Fish MB Fisher M Oomen-Hajagos J Thomsen GH Grainger RM Simple and efficient CRISPR/Cas9-mediated targeted mutagenesis in Xenopus tropicalis Genesis 2013 51 835 843 24123613
29 Jinek M Jiang F Taylor DW Structures of Cas9 endonucleases reveal RNA-mediated conformational activation Science 2014 343 1247997 24505130
30 Nishimasu H Ran FA Hsu PD Crystal structure of Cas9 in complex with guide RNA and target DNA Cell 2014 156 935 949 24529477
31 Wang T Wei JJ Sabatini DM Lander ES Genetic screens in human cells using the CRISPR-Cas9 system Science 2014 343 80 84 24336569
32 Shalem O Sanjana NE Hartenian E Genome-scale CRISPR-Cas9 knockout screening in human cells Science 2014 343 84 87 24336571
33 Niu Y Shen B Cui Y Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos Cell 2014 156 836 843 24486104
34 Wagner JC Platt RJ Goldfless SJ Zhang F Niles JC Efficient CRISPR-Cas9-mediated genome editing in Plasmodium falciparum Nat Methods 2014 11 915 918 25108687
35 Liang P Xu Y Zhang X CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes Protein Cell 2015 6 363 372 25894090
36 Cleto S Jensen JV Wendisch VF Lu TK Corynebacterium glutamicum metabolic engineering with CRISPR interference (CRISPRi) ACS Synth Biol 2016 5 375 385 26829286
37 Wright AV Nunez JK Doudna JA Biology and applications of CRISPR systems: harnessing nature’s toolbox for genome engineering Cell 2016 164 29 44 26771484
38 Shmakov S Abudayyeh OO Makarova KS Discovery and functional characterization of diverse class 2 CRISPR-Cas systems Mol Cell 2015 60 385 397 26593719
39 Doudna JA Charpentier E Genome editing: the new frontier of genome engineering with CRISPR-Cas9 Science 2014 346 1258096 25430774
40 Makarova KS Haft DH Barrangou R Evolution and classification of the CRISPR-Cas systems Nat Rev Microbiol 2011 9 467 477 21552286
41 Makarova KS Wolf YI Alkhnbashi OS An updated evolutionary classification of CRISPR-Cas systems Nat Rev Microbiol 2015 13 722 736 26411297
42 Lander ES The heroes of CRISPR Cell 2016 164 18 28 26771483
43 Charpentier E Richter H van der Oost J White MF Biogenesis pathways of RNA guides in archaeal and bacterial CRISPR-Cas adaptive immunity FEMS Microbiol Rev 2015 39 428 441 25994611
44 Kim H Kim JS A guide to genome engineering with programmable nucleases Nat Rev Genet 2014 15 321 334 24690881
45 Charpentier E Doudna JA Biotechnology: rewriting a genome Nature 2013 495 50 51 23467164
46 Cox DB Platt RJ Zhang F Therapeutic genome editing: prospects and challenges Nat Med 2015 21 121 131 25654603
47 Silva G Poirot L Galetto R Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy Curr Gene Ther 2011 11 11 27 21182466
48 Urnov FD Rebar EJ Holmes MC Zhang HS Gregory PD Genome editing with engineered zinc finger nucleases Nat Rev Genet 2010 11 636 646 20717154
49 Christian M Cermak T Doyle EL Targeting DNA double-strand breaks with TAL effector nucleases Genetics 2010 186 757 761 20660643
50 Maeder ML Thibodeau-Beganny S Osiak A Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification Mol Cell 2008 31 294 301 18657511
51 Moscou MJ Bogdanove AJ A simple cipher governs DNA recognition by TAL effectors Science 2009 326 1501 19933106
52 Joung JK Sander JD TALENs: a widely applicable technology for targeted genome editing Nat Rev Mol Cell Biol 2013 14 49 55 23169466
53 Prakash V Moore M Yanez-Munoz RJ Current progress in therapeutic gene editing for monogenic diseases Mol Ther 2016 24 465 474 26765770
54 Ousterout DG Perez-Pinera P Thakore PI Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients Mol Ther 2013 21 1718 1726 23732986
55 Benabdallah BF Duval A Rousseau J Targeted gene addition of microdystrophin in mice skeletal muscle via human myoblast transplantation Mol Ther Nucleic Acids 2013 2 e68 23360951
56 Ousterout DG Kabadi AM Thakore PI Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases Mol Ther 2015 23 523 532 25492562
57 Ousterout DG Kabadi AM Thakore PI Majoros WH Reddy TE Gersbach CA Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy Nat Commun 2015 6 6244 25692716
58 Voit RA Hendel A Pruett-Miller SM Porteus MH Nuclease-mediated gene editing by homologous recombination of the human globin locus Nucleic Acids Res 2014 42 1365 1378 24157834
59 Hu W Kaminski R Yang F RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection Proc Natl Acad Sci U S A 2014 111 11461 11466 25049410
60 Lin SR Yang HC Kuo YT The CRISPR/Cas9 system facilitates clearance of the intrahepatic HBV templates in vivo Mol Ther Nucleic Acids 2014 3 e186 25137139
61 Kennedy EM Bassit LC Mueller H Suppression of hepatitis B virus DNA accumulation in chronically infected cells using a bacterial CRISPR/Cas RNA-guided DNA endonuclease Virology 2015 476 196 205 25553515
62 Seeger C Sohn JA Targeting hepatitis B virus with CRISPR/Cas9 Mol Ther Nucleic Acids 2014 3 e216 25514649
63 Ramanan V Shlomai A Cox DB CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus Sci Rep 2015 5 10833 26035283
64 Zhen S Hua L Liu YH Harnessing the clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated Cas9 system to disrupt the hepatitis B virus Gene Ther 2015 22 404 412 25652100
65 Dong C Qu L Wang H Wei L Dong Y Xiong S Targeting hepatitis B virus cccDNA by CRISPR/Cas9 nuclease efficiently inhibits viral replication Antiviral Res 2015 118 110 117 25843425
66 Liu X Hao R Chen S Guo D Chen Y Inhibition of hepatitis B virus by the CRISPR/Cas9 system via targeting the conserved regions of the viral genome J Gen Virol 2015 96 2252 2261 25904148
67 Zhen S Hua L Takahashi Y Narita S Liu YH Li Y In vitro and in vivo growth suppression of human papillomavirus 16-positive cervical cancer cells by CRISPR/Cas9 Biochem Biophys Res Commun 2014 450 1422 1426 25044113
68 Kennedy EM Kornepati AV Goldstein M Inactivation of the human papillomavirus E6 or E7 gene in cervical carcinoma cells by using a bacterial CRISPR/Cas RNA-guided endonuclease J Virol 2014 88 11965 11972 25100830
69 Yu L Wang X Zhu D Disruption of human papillomavirus 16 E6 gene by clustered regularly interspaced short palindromic repeat/Cas system in human cervical cancer cells Onco Targets Ther 2015 8 37 44 25565864
70 Hu Z Yu L Zhu D Disruption of HPV16-E7 by CRISPR/Cas system induces apoptosis and growth inhibition in HPV16 positive human cervical cancer cells Biomed Res Int 2014 2014 612823 25136604
71 Lee CM Flynn R Hollywood JA Scallan MF Harrison PT Correction of the deltaF508 mutation in the cystic fibrosis transmembrane conductance regulator gene by zinc-finger nuclease homology-directed repair Biores Open Access 2012 1 99 108 23514673
72 Li XB Chen J Deng MJ Wang F Du ZW Zhang JW Zinc finger protein HZF1 promotes K562 cell proliferation by interacting with and inhibiting INCA1 Mol Med Rep 2011 4 1131 1137 21874239
73 Lombardo A Genovese P Beausejour CM Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery Nat Biotechnol 2007 25 1298 1306 17965707
74 Urnov FD Miller JC Lee YL Highly efficient endogenous human gene correction using designed zinc-finger nucleases Nature 2005 435 646 651 15806097
75 Matsubara Y Chiba T Kashimada K Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus Sci Rep 2014 4 5043 24853770
76 Genovese P Schiroli G Escobar G Targeted genome editing in human repopulating haematopoietic stem cells Nature 2014 510 235 240 24870228
77 Osborn MJ Starker CG McElroy AN TALEN-based gene correction for epidermolysis bullosa Mol Ther 2013 21 1151 1159 23546300
78 Nelson CE Hakim CH Ousterout DG In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy Science 2016 351 403 407 26721684
79 Tabebordbar M Zhu K Cheng JK In vivo gene editing in dystrophic mouse muscle and muscle stem cells Science 2016 351 407 411 26721686
80 Long C Amoasii L Mireault AA Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy Science 2016 351 400 403 26721683
81 Wu Y Zhou H Fan X Correction of a genetic disease by CRISPR-Cas9-mediated gene editing in mouse spermatogonial stem cells Cell Res 2015 25 67 79 25475058
82 Li H Haurigot V Doyon Y In vivo genome editing restores haemostasis in a mouse model of haemophilia Nature 2011 475 217 221 21706032
83 Anguela XM Sharma R Doyon Y Robust ZFN-mediated genome editing in adult hemophilic mice Blood 2013 122 3283 3287 24085764
84 Sharma R Anguela XM Doyon Y In vivo genome editing of the albumin locus as a platform for protein replacement therapy Blood 2015 126 1777 1784 26297739
85 Yin H Xue W Chen S Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype Nat Biotechnol 2014 32 551 553 24681508
86 Long C McAnally JR Shelton JM Mireault AA Bassel-Duby R Olson EN Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA Science 2014 345 1184 1188 25123483
87 Xu L Park KH Zhao L CRISPR-mediated genome editing restores dystrophin expression and function in mdx mice Mol Ther 2016 24 564 569 26449883
88 Ding Q Strong A Patel KM Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing Circ Res 2014 115 488 492 24916110
89 Li HL Fujimoto N Sasakawa N Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9 Stem Cell Reports 2015 4 143 154 25434822
90 Park CY Kim J Kweon J Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs Proc Natl Acad Sci U S A 2014 111 9253 9258 24927536
91 Park CY Kim DH Son JS Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9 Cell Stem Cell 2015 17 213 220 26212079
92 Hoban MD Cost GJ Mendel MC Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells Blood 2015 125 2597 2604 25733580
93 Sebastiano V Maeder ML Angstman JF In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases Stem Cells 2011 29 1717 1726 21898685
94 Sun N Zhao H Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs Biotechnol Bioeng 2014 111 1048 1053 23928856
95 Huang X Wang Y Yan W Production of gene-corrected adult beta globin protein in human erythrocytes differentiated from patient iPSCs after genome editing of the sickle point mutation Stem Cells 2015 33 1470 1479 25702619
96 Xie F Ye L Chang JC Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac Genome Res 2014 24 1526 1533 25096406
97 Ma N Shan Y Liao B Factor-induced reprogramming and zinc finger nuclease-aided gene targeting cause different genome instability in beta-thalassemia induced pluripotent stem cells (iPSCs) J Biol Chem 2015 290 12079 12089 25795783
98 Ye L Wang J Beyer AI Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Delta32 mutation confers resistance to HIV infection Proc Natl Acad Sci U S A 2014 111 9591 9596 24927590
99 Wilen CB Wang J Tilton JC Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases PLoS Pathog 2011 7 e1002020 21533216
100 Sebastiano V Zhen HH Haddad B Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa Sci Transl Med 2014 6 264ra163
101 Crane AM Kramer P Bui JH Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells Stem Cell Reports 2015 4 569 577 25772471
102 Sargent RG Suzuki S Gruenert DC Nuclease-mediated double-strand break (DSB) enhancement of small fragment homologous recombination (SFHR) gene modification in human-induced pluripotent stem cells (hiPSCs) Methods Mol Biol 2014 1114 279 290 24557910
103 Firth AL Menon T Parker GS Functional gene correction for cystic fibrosis in lung epithelial cells generated from patient iPSCs Cell Rep 2015 12 1385 1390 26299960
104 Schwank G Koo BK Sasselli V Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients Cell Stem Cell 2013 13 653 658 24315439
105 Suzuki S Sargent RG Illek B TALENs facilitate single-step seamless SDF correction of F508del CFTR in airway epithelial submucosal gland cell-derived CF-iPSCs Mol Ther Nucleic Acids 2016 5 e273 26730810
106 Vierstra J Reik A Chang KH Functional footprinting of regulatory DNA Nat Methods 2015 12 927 930 26322838
107 Canver MC Smith EC Sher F BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis Nature 2015 527 192 197 26375006
108 Rahman SH Kuehle J Reimann C Rescue of DNAPK signaling and T-cell differentiation by targeted genome editing in a prkdc deficient iPSC disease model PLoS Genet 2015 11 e1005239 26000857
109 Menon T Firth AL Scripture-Adams DD Lymphoid regeneration from gene-corrected SCID-X1 subject-derived iPSCs Cell Stem Cell 2015 16 367 372 25772073
110 Holt N Wang J Kim K Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo Nat Biotechnol 2010 28 839 847 20601939
111 Li L Krymskaya L Wang J Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases Mol Ther 2013 21 1259 1269 23587921
112 Nathwani AC Tuddenham EG Rangarajan S Adenovirus-associated virus vector-mediated gene transfer in hemophilia B N Engl J Med 2011 365 2357 2365 22149959
113 Nathwani AC Reiss UM Tuddenham EG Longterm safety and efficacy of factor IX gene therapy in hemophilia B N Engl J Med 2014 371 1994 2004 25409372
114 Hutter G Nowak D Mossner M Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation N Engl J Med 2009 360 692 698 19213682
115 Tebas P Stein D Tang WW Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV N Engl J Med 2014 370 901 910 24597865
116 Badia R Pauls E Riveira-Munoz E Clotet B Este JA Ballana E Zinc finger endonuclease targeting PSIP1 inhibits HIV-1 integration Antimicrob Agents Chemother 2014 58 4318 4327 24820090
117 Fadel HJ Morrison JH Saenz DT TALEN knockout of the PSIP1 gene in human cells: analyses of HIV-1 replication and allosteric integrase inhibitor mechanism J Virol 2014 88 9704 9717 24942577
118 White MK Khalili K CRISPR/Cas9 and cancer targets: future possibilities and present challenges Oncotarget 2016 7 12305 12317 26840090
119 Brown MT Cooper JA Regulation, substrates and functions of src Biochim Biophys Acta 1996 1287 121 149 8672527
120 Choi PS Meyerson M Targeted genomic rearrangements using CRISPR/Cas technology Nat Commun 2014 5 3728 24759083
121 Chen C Liu Y Rappaport AR MLL3 is a haploinsufficient 7q tumor suppressor in acute myeloid leukemia Cancer Cell 2014 25 652 665 24794707
122 Xue W Chen S Yin H CRISPR-mediated direct mutation of cancer genes in the mouse liver Nature 2014 514 380 384 25119044
123 Chiou SH Winters IP Wang J Pancreatic cancer modeling using retrograde viral vector delivery and in vivo CRISPR/Cas9-mediated somatic genome editing Genes Dev 2015 29 1576 1585 26178787
124 Tu Z Yang W Yan S Guo X Li XJ CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases Mol Neurodegener 2015 10 35 26238861
125 Hsu PD Lander ES Zhang F Development and applications of CRISPR-Cas9 for genome engineering Cell 2014 157 1262 1278 24906146
126 Jusiak B Cleto S Perez-Pinera P Lu TK Engineering synthetic gene circuits in living cells with CRISPR technology Trends Biotechnol 2016 34 535 547 26809780
127 Wang Y Cheng X Shan Q Simultaneous editing of three homoeoalleles in hexaploid bread wheat confers heritable resistance to powdery mildew Nat Biotechnol 2014 32 947 951 25038773
128 Zhou Y Zhu S Cai C High-throughput screening of a CRISPR/Cas9 library for functional genomics in human cells Nature 2014 509 487 491 24717434
129 Diecke S Jung SM Lee J Ju JH Recent technological updates and clinical applications of induced pluripotent stem cells Korean J Intern Med 2014 29 547 557 25228828
130 Musunuru K Genome editing of human pluripotent stem cells to generate human cellular disease models Dis Model Mech 2013 6 896 904 23751357
131 Grobarczyk B Franco B Hanon K Malgrange B Generation of isogenic human iPS cell line precisely corrected by genome editing using the CRISPR/Cas9 system Stem Cell Rev 2015 11 774 787 26059412
132 Orqueda AJ Gimenez CA Pereyra-Bonnet F iPSCs: a minireview from bench to bed, including organoids and the CRISPR system Stem Cells Int 2016 2016 5934782 26880972
133 Maruyama T Dougan SK Truttmann MC Bilate AM Ingram JR Ploegh HL Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining Nat Biotechnol 2015 33 538 542 25798939
134 Chu VT Weber T Wefers B Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells Nat Biotechnol 2015 33 543 548 25803306
135 Nakade S Tsubota T Sakane Y Microhomology-mediated end-joining-dependent integration of donor DNA in cells and animals using TALENs and CRISPR/Cas9 Nat Commun 2014 5 5560 25410609
136 Sakuma T Nakade S Sakane Y Suzuki KT Yamamoto T MMEJ-assisted gene knock-in using TALENs and CRISPR-Cas9 with the PITCh systems Nat Protoc 2016 11 118 133 26678082
137 Ledford H Bacteria yield new gene cutter Nature 2015 526 17 26432219
138 Hsu PD Scott DA Weinstein JA DNA targeting specificity of RNA-guided Cas9 nucleases Nat Biotechnol 2013 31 827 832 23873081
139 Mali P Aach J Stranges PB CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering Nat Biotechnol 2013 31 833 838 23907171
140 Ran FA Hsu PD Lin CY Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity Cell 2013 154 1380 1389 23992846
141 Fu Y Sander JD Reyon D Cascio VM Joung JK Improving CRISPR-Cas nuclease specificity using truncated guide RNAs Nat Biotechnol 2014 32 279 284 24463574
142 Guilinger JP Thompson DB Liu DR Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification Nat Biotechnol 2014 32 577 582 24770324
143 Tsai SQ Wyvekens N Khayter C Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing Nat Biotechnol 2014 32 569 576 24770325
144 Hastie E Samulski RJ Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success: a personal perspective Hum Gene Ther 2015 26 257 265 25807962
145 Moore R Spinhirne A Lai MJ CRISPR-based self-cleaving mechanism for controllable gene delivery in human cells Nucleic Acids Res 2015 43 1297 1303 25527740
146 Kormann MS Hasenpusch G Aneja MK Expression of therapeutic proteins after delivery of chemically modified mRNA in mice Nat Biotechnol 2011 29 154 157 21217696
147 Zuris JA Thompson DB Shu Y Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo Nat Biotechnol 2015 33 73 80 25357182
해당 논문의 주제분야에서 활용도가 높은 상위 5개 콘텐츠를 보여줍니다.
더보기 버튼을 클릭하시면 더 많은 관련자료를 살펴볼 수 있습니다.
*원문 PDF 파일 및 링크정보가 존재하지 않을 경우 KISTI DDS 시스템에서 제공하는 원문복사서비스를 사용할 수 있습니다.
오픈액세스 학술지에 출판된 논문
※ AI-Helper는 부적절한 답변을 할 수 있습니다.