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A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. <i>Science<\/i>, <b>337<\/b>, 816\u2013821 (2012).","DOI":"10.1126\/science.1225829"},{"key":"2","doi-asserted-by":"crossref","unstructured":"2) Garneau JE, Dupuis M-\u00c8, Villion M, Romero DA, Barrangou R, Boyaval P, Fremaux C, Horvath P, Magad\u00e1n AH, Moineau S. The CRISPR\/Cas bacterial immune system cleaves bacteriophage and plasmid DNA. <i>Nature<\/i>, <b>468<\/b>, 67\u201371 (2010).","DOI":"10.1038\/nature09523"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3) Komor AC, Badran AH, Liu DR. CRISPR-based technologies for the manipulation of eukaryotic genomes. <i>Cell<\/i>, <b>168<\/b>, 20\u201336 (2017).","DOI":"10.1016\/j.cell.2016.10.044"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4) Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI, Porteus MH. CRISPR\/Cas9 \u03b2-globin gene targeting in human haematopoietic stem cells. <i>Nature<\/i>, <b>539<\/b>, 384\u2013389 (2016).","DOI":"10.1038\/nature20134"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5) Ohmori T, Nagao Y, Mizukami H, Sakata A, Muramatsu S-I, Ozawa K, Tominaga S-I, Hanazono Y, Nishimura S, Nureki O, Sakata Y. CRISPR\/Cas9-mediated genome editing <i>via<\/i> postnatal administration of AAV vector cures haemophilia B mice. <i>Sci. Rep.<\/i>, <b>7<\/b>, 4159 (2017).","DOI":"10.1038\/s41598-017-04625-5"},{"key":"6","doi-asserted-by":"crossref","unstructured":"6) Park C-Y, Kim DH, Son JS, Sung JJ, Lee J, Bae S, Kim J-H, Kim D-W, Kim J-S. Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9. <i>Cell Stem Cell<\/i>, <b>17<\/b>, 213\u2013220 (2015).","DOI":"10.1016\/j.stem.2015.07.001"},{"key":"7","doi-asserted-by":"crossref","unstructured":"7) Huang Z, Tomitaka A, Raymond A, Nair M. Current application of CRISPR\/Cas9 gene-editing technique to eradication of HIV\/AIDS. <i>Gene Ther.<\/i>, <b>24<\/b>, 377\u2013384 (2017).","DOI":"10.1038\/gt.2017.35"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8) Aravalli RN, Steer CJ. CRISPR\/Cas9 therapeutics for liver diseases. <i>J. Cell. Biochem.<\/i>, <b>119<\/b>, 4265\u20134278 (2018).","DOI":"10.1002\/jcb.26627"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9) Dandri M, Burda MR, Torok E, Pollok JM, Iwanska A, Sommer G, Rogiers X, Rogler CE, Gupta S, Will H, Greten H, Petersen J. Repopulation of mouse liver with human hepatocytes and <i>in vivo<\/i> infection with hepatitis B virus. <i>Hepatology<\/i>, <b>33<\/b>, 981\u2013988 (2001).","DOI":"10.1053\/jhep.2001.23314"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10) Mercer DF, Schiller DE, Elliott JF, Douglas DN, Hao C, Rinfret A, Addison WR, Fischer KP, Churchill TA, Lakey JR, Tyrrell DL, Kneteman NM. Hepatitis C virus replication in mice with chimeric human livers. <i>Nat. Med.<\/i>, <b>7<\/b>, 927\u2013933 (2001).","DOI":"10.1038\/90968"},{"key":"11","doi-asserted-by":"crossref","unstructured":"11) Tateno C, Yoshizane Y, Saito N, Kataoka M, Utoh R, Yamasaki C, Tachibana A, Soeno Y, Asahina K, Hino H, Asahara T, Yokoi T, Furukawa T, Yoshizato K. Near completely humanized liver in mice shows human-type metabolic responses to drugs. <i>Am. J. Pathol.<\/i>, <b>165<\/b>, 901\u2013912 (2004).","DOI":"10.1016\/S0002-9440(10)63352-4"},{"key":"12","doi-asserted-by":"crossref","unstructured":"12) Zetsche B, Gootenberg JS, Abudayyeh OO, Slaymaker IM, Makarova KS, Essletzbichler P, Volz SE, Joung J, van der Oost J, Regev A, Koonin EV, Zhang F. Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system. <i>Cell<\/i>, <b>163<\/b>, 759\u2013771 (2015).","DOI":"10.1016\/j.cell.2015.09.038"},{"key":"13","doi-asserted-by":"crossref","unstructured":"13) Schmidt F, Grimm D. CRISPR genome engineering and viral gene delivery: a case of mutual attraction. <i>Biotechnol. J.<\/i>, <b>10<\/b>, 258\u2013272 (2015).","DOI":"10.1002\/biot.201400529"},{"key":"14","doi-asserted-by":"crossref","unstructured":"14) Zetsche B, Heidenreich M, Mohanraju P, Fedorova I, Kneppers J, DeGennaro EM, Winblad N, Choudhury SR, Abudayyeh OO, Gootenberg JS, Wu WY, Scott DA, Severinov K, van der Oost J, Zhang F. Multiplex gene editing by CRISPR-Cpf1 through autonomous processing of a single crRNA array. <i>Nat. Biotechnol.<\/i>, <b>35<\/b>, 31\u201334 (2017).","DOI":"10.1038\/nbt.3737"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15) Sakurai F, Kawabata K, Koizumi N, Inoue N, Okabe M, Yamaguchi T, Hayakawa T, Mizuguchi H. Adenovirus serotype 35 vector-mediated transduction into human CD46-transgenic mice. <i>Gene Ther.<\/i>, <b>13<\/b>, 1118\u20131126 (2006).","DOI":"10.1038\/sj.gt.3302749"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16) Takayama K, Igai K, Hagihara Y, Hashimoto R, Hanawa M, Sakuma T, Tachibana M, Sakurai F, Yamamoto T, Mizuguchi H. Highly efficient biallelic genome editing of human ES\/iPS cells using a CRISPR\/Cas9 or TALEN system. <i>Nucleic Acids Res.<\/i>, <b>45<\/b>, 5198\u20135207 (2017).","DOI":"10.1093\/nar\/gkx130"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17) Mizuguchi H, Kay MA. A simple method for constructing E1- and E1\/E4-deleted recombinant adenoviral vectors. <i>Hum. Gene Ther.<\/i>, <b>10<\/b>, 2013\u20132017 (1999).","DOI":"10.1089\/10430349950017374"},{"key":"18","doi-asserted-by":"crossref","unstructured":"18) Machitani M, Sakurai F, Wakabayashi K, Nakatani K, Takayama K, Tachibana M, Mizuguchi H. Inhibition of CRISPR\/Cas9-mediated genome engineering by a type I interferon-induced reduction in guide RNA expression. <i>Biol. Pharm. Bull.<\/i>, <b>40<\/b>, 272\u2013277 (2017).","DOI":"10.1248\/bpb.b16-00700"},{"key":"19","doi-asserted-by":"crossref","unstructured":"19) Tashiro K, Inamura M, Kawabata K, Sakurai F, Yamanishi K, Hayakawa T, Mizuguchi H. Efficient adipocyte and osteoblast differentiation from mouse induced pluripotent stem cells by adenoviral transduction. <i>Stem Cells<\/i>, <b>27<\/b>, 1802\u20131811 (2009).","DOI":"10.1002\/stem.108"},{"key":"20","doi-asserted-by":"crossref","unstructured":"20) Mizuguchi H, Kay MA. Efficient construction of a recombinant adenovirus vector by an improved <i>in vitro<\/i> ligation method. <i>Hum. Gene Ther.<\/i>, <b>9<\/b>, 2577\u20132583 (1998).","DOI":"10.1089\/hum.1998.9.17-2577"},{"key":"21","doi-asserted-by":"crossref","unstructured":"21) Mukai E, Fujimoto S, Sakurai F, Kawabata K, Yamashita M, Inagaki N, Mizuguchi H. Efficient gene transfer into murine pancreatic islets using adenovirus vectors. <i>J. Control. Release<\/i>, <b>119<\/b>, 136\u2013141 (2007).","DOI":"10.1016\/j.jconrel.2007.01.012"},{"key":"22","doi-asserted-by":"crossref","unstructured":"22) Maizel JVJ Jr, White DO, Scharff MD. The polypeptides of adenovirus. I. Evidence for multiple protein components in the virion and a comparison of types 2, 7A, and 12. <i>Virology<\/i>, <b>36<\/b>, 115\u2013125 (1968).","DOI":"10.1016\/0042-6822(68)90121-9"},{"key":"23","doi-asserted-by":"crossref","unstructured":"23) Lombardo A, Cesana D, Genovese P, Di Stefano B, Provasi E, Colombo DF, Neri M, Magnani Z, Cantore A, Lo Riso P, Damo M, Pello OM, Holmes MC, Gregory PD, Gritti A, Broccoli V, Bonini C, Naldini L. Site-specific integration and tailoring of cassette design for sustainable gene transfer. <i>Nat. Methods<\/i>, <b>8<\/b>, 861\u2013869 (2011).","DOI":"10.1038\/nmeth.1674"},{"key":"24","doi-asserted-by":"crossref","unstructured":"24) Ordovas L, Boon R, Pistoni M, Chen Y, Wolfs E, Guo W, Sambathkumar R, Bobis-Wozowicz S, Helsen N, Vanhove J, Berckmans P, Cai Q, Vanuytsel K, Eggermont K, Vanslembrouck V, Schmidt BZ, Raitano S, Van Den Bosch L, Nahmias Y, Cathomen T, Struys T, Verfaillie CM. Efficient recombinase-mediated cassette exchange in hPSCs to study the hepatocyte lineage reveals AAVS1 locus-mediated transgene inhibition. <i>Stem Cell Reports<\/i>, <b>5<\/b>, 918\u2013931 (2015).","DOI":"10.1016\/j.stemcr.2015.09.004"},{"key":"25","doi-asserted-by":"crossref","unstructured":"25) DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Steine EJ, Miller JC, Tam P, Bartsevich VV, Meng X, Rupniewski I, Gopalan SM, Sun HC, Pitz KJ, Rock JM, Zhang L, Davis GD, Rebar EJ, Cheeseman IM, Yamamoto KR, Sabatini DM, Jaenisch R, Gregory PD, Urnov FD. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome. <i>Genome Res.<\/i>, <b>20<\/b>, 1133\u20131142 (2010).","DOI":"10.1101\/gr.106773.110"},{"key":"26","doi-asserted-by":"crossref","unstructured":"26) Bae S, Park J, Kim J-S. Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases. <i>Bioinformatics<\/i>, <b>30<\/b>, 1473\u20131475 (2014).","DOI":"10.1093\/bioinformatics\/btu048"},{"key":"27","doi-asserted-by":"crossref","unstructured":"27) Kim D, Kim J, Hur JK, Been KW, Yoon S, Kim J-S. Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells. <i>Nat. Biotechnol.<\/i>, <b>34<\/b>, 863\u2013868 (2016).","DOI":"10.1038\/nbt.3609"},{"key":"28","doi-asserted-by":"crossref","unstructured":"28) Shimizu K, Sakurai F, Machitani M, Katayama K, Mizuguchi H. Quantitative analysis of the leaky expression of adenovirus genes in cells transduced with a replication-incompetent adenovirus vector. <i>Mol. Pharm.<\/i>, <b>8<\/b>, 1430\u20131435 (2011).","DOI":"10.1021\/mp200121z"},{"key":"29","doi-asserted-by":"crossref","unstructured":"29) Zhang Y, Long C, Li H, McAnally JR, Baskin KK, Shelton JM, Bassel-Duby R, Olson EN. CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice. <i>Sci. Adv.<\/i>, <b>3<\/b>, e1602814 (2017).","DOI":"10.1126\/sciadv.1602814"},{"key":"30","doi-asserted-by":"crossref","unstructured":"30) Shimizu K, Sakurai F, Tomita K, Nagamoto Y, Nakamura S-I, Katayama K, Tachibana M, Kawabata K, Mizuguchi H. Suppression of leaky expression of adenovirus genes by insertion of microRNA-targeted sequences in the replication-incompetent adenovirus vector genome. <i>Mol. Ther. Methods Clin. Dev.<\/i>, <b>1<\/b>, 14035 (2014).","DOI":"10.1038\/mtm.2014.35"},{"key":"31","doi-asserted-by":"crossref","unstructured":"31) Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HCJ, Rice CM, Srivastava A, de Jong YP, Herzog RW. Superior <i>in vivo<\/i> transduction of human hepatocytes using engineered AAV3 capsid. <i>Mol. Ther.<\/i>, <b>24<\/b>, 1042\u20131049 (2016).","DOI":"10.1038\/mt.2016.61"},{"key":"32","doi-asserted-by":"crossref","unstructured":"32) Khetani SR, Bhatia SN. Microscale culture of human liver cells for drug development. <i>Nat. Biotechnol.<\/i>, <b>26<\/b>, 120\u2013126 (2008).","DOI":"10.1038\/nbt1361"},{"key":"33","doi-asserted-by":"crossref","unstructured":"33) Godoy P, Widera A, Schmidt-Heck W, Campos G, Meyer C, Cadenas C, Reif R, Stober R, Hammad S, Putter L, Gianmoena K, Marchan R, Ghallab A, Edlund K, Nussler A, Thasler WE, Damm G, Seehofer D, Weiss TS, Dirsch O, Dahmen U, Gebhardt R, Chaudhari U, Meganathan K, Sachinidis A, Kelm J, Hofmann U, Zahedi RP, Guthke R, Bluthgen N, Dooley S, Hengstler JG. Gene network activity in cultivated primary hepatocytes is highly similar to diseased mammalian liver tissue. <i>Arch. Toxicol.<\/i>, <b>90<\/b>, 2513\u20132529 (2016).","DOI":"10.1007\/s00204-016-1761-4"},{"key":"34","doi-asserted-by":"crossref","unstructured":"34) Brunetti-Pierri N, Ng P. Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models. <i>Virus Genes<\/i>, <b>53<\/b>, 684\u2013691 (2017).","DOI":"10.1007\/s11262-017-1471-x"},{"key":"35","doi-asserted-by":"crossref","unstructured":"35) Piccolo P, Brunetti-Pierri N. Challenges and prospects for helper-dependent adenoviral vector-mediated gene therapy. <i>Biomedicines<\/i>, <b>2<\/b>, 132\u2013148 (2014).","DOI":"10.3390\/biomedicines2020132"},{"key":"36","doi-asserted-by":"crossref","unstructured":"36) Suzuki K, Tsunekawa Y, Hernandez-Benitez R, Wu J, Zhu J, Kim EJ, Hatanaka F, Yamamoto M, Araoka T, Li Z, Kurita M, Hishida T, Li M, Aizawa E, Guo S, Chen S, Goebl A, Soligalla RD, Qu J, Jiang T, Fu X, Jafari M, Esteban CR, Berggren WT, Lajara J, Nunez-Delicado E, Guillen P, Campistol JM, Matsuzaki F, Liu G-H, Magistretti P, Zhang K, Callaway EM, Zhang K, Belmonte JCI. <i>In vivo<\/i> genome editing <i>via<\/i> CRISPR\/Cas9 mediated homology-independent targeted integration. <i>Nature<\/i>, <b>540<\/b>, 144\u2013149 (2016).","DOI":"10.1038\/nature20565"},{"key":"37","doi-asserted-by":"crossref","unstructured":"37) Orthwein A, Noordermeer SM, Wilson MD, Landry S, Enchev RI, Sherker A, Munro M, Pinder J, Salsman J, Dellaire G, Xia B, Peter M, Durocher D. A mechanism for the suppression of homologous recombination in G1 cells. <i>Nature<\/i>, <b>528<\/b>, 422\u2013426 (2015).","DOI":"10.1038\/nature16142"}],"container-title":["Biological and Pharmaceutical Bulletin"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/bpb\/41\/7\/41_b18-00222\/_pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2018,7,7]],"date-time":"2018-07-07T00:19:45Z","timestamp":1530922785000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/bpb\/41\/7\/41_b18-00222\/_article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,7,1]]},"references-count":37,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2018]]}},"URL":"https:\/\/doi.org\/10.1248\/bpb.b18-00222","relation":{},"ISSN":["0918-6158","1347-5215"],"issn-type":[{"value":"0918-6158","type":"print"},{"value":"1347-5215","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,7,1]]}}}