{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T21:02:39Z","timestamp":1792098159957,"version":"4.4.0"},"reference-count":38,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2016,3,10]],"date-time":"2016-03-10T00:00:00Z","timestamp":1457568000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2016,3,10]],"date-time":"2016-03-10T00:00:00Z","timestamp":1457568000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"State Key Development Programs of China","award":["2012CB910400"],"award-info":[{"award-number":["2012CB910400"]}]},{"name":"State Key Development Programs of China","award":["2012CB966502"],"award-info":[{"award-number":["2012CB966502"]}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["31371455"],"award-info":[{"award-number":["31371455"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81202104"],"award-info":[{"award-number":["81202104"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81330049"],"award-info":[{"award-number":["81330049"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81060016"],"award-info":[{"award-number":["81060016"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81460034"],"award-info":[{"award-number":["81460034"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["81260032"],"award-info":[{"award-number":["81260032"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["31140021"],"award-info":[{"award-number":["31140021"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"name":"International Science & Technology Cooperation Program of China","award":["2014DFA30180"],"award-info":[{"award-number":["2014DFA30180"]}]},{"name":"Shanghai Municipal Commission for Science and Technology","award":["14140900300"],"award-info":[{"award-number":["14140900300"]}]},{"name":"Shanghai Municipal Commission for Science and Technology","award":["15JC1400201"],"award-info":[{"award-number":["15JC1400201"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["EMBO Mol Med"],"published-print":{"date-parts":[[2016,5,1]]},"DOI":"10.15252\/emmm.201506039","type":"journal-article","created":{"date-parts":[[2016,3,10]],"date-time":"2016-03-10T21:09:51Z","timestamp":1457644191000},"page":"477-488","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":161,"title":["CRISPR\/Cas9\u2010mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse"],"prefix":"10.1038","volume":"8","author":[{"given":"Yuting","family":"Guan","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanlin","family":"Ma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qi","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhenliang","family":"Sun","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lie","family":"Ma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lijuan","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liren","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Zeng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanjiao","family":"Shao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuting","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ning","family":"Ma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wenqing","family":"Lu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kewen","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Honghui","family":"Han","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanhong","family":"Yu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuanhua","family":"Huang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mingyao","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dali","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2016,3,10]]},"reference":[{"key":"BFEMMM201506039_CR1","doi-asserted-by":"crossref","first-page":"3283","DOI":"10.1182\/blood-2013-04-497354","volume":"122","author":"XM Anguela","year":"2013","unstructured":"Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S et\u00a0al (2013) Robust ZFN\u2010mediated genome editing in adult hemophilic mice. Blood 122: 3283\u20133287","journal-title":"Blood"},{"key":"BFEMMM201506039_CR2","doi-asserted-by":"crossref","first-page":"360","DOI":"10.1038\/nature13864","volume":"517","author":"A Barzel","year":"2015","unstructured":"Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM et\u00a0al (2015) Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature 517: 360\u2013364","journal-title":"Nature"},{"key":"BFEMMM201506039_CR3","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1089\/10430340460732445","volume":"15","author":"N Brunetti\u2010Pierri","year":"2004","unstructured":"Brunetti\u2010Pierri N, Palmer DJ, Beaudet AL, Carey KD, Finegold M, Ng P (2004) Acute toxicity after high\u2010dose systemic injection of helper\u2010dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 15: 35\u201346","journal-title":"Hum Gene Ther"},{"key":"BFEMMM201506039_CR4","doi-asserted-by":"crossref","first-page":"25393","DOI":"10.1074\/jbc.M105432200","volume":"277","author":"YJ Chang","year":"2002","unstructured":"Chang YJ, Wu HL, Hamaguchi N, Hsu YC, Lin SW (2002) Identification of functionally important residues of the epidermal growth factor\u20102 domain of factor IX by alanine\u2010scanning mutagenesis. Residues Asn(89)\u2010Gly(93) are critical for binding factor VIIIa. J Biol Chem 277: 25393\u201325399","journal-title":"J Biol Chem"},{"key":"BFEMMM201506039_CR5","doi-asserted-by":"crossref","first-page":"819","DOI":"10.1126\/science.1231143","volume":"339","author":"L Cong","year":"2013","unstructured":"Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, Hsu PD, Wu X, Jiang W, Marraffini LA et\u00a0al (2013) Multiplex genome engineering using CRISPR\/Cas systems. Science 339: 819\u2013823","journal-title":"Science"},{"key":"BFEMMM201506039_CR6","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1089\/hum.2013.2527","volume":"25","author":"RG Crystal","year":"2014","unstructured":"Crystal RG (2014) Adenovirus: the first effective in\u00a0vivo gene delivery vector. Hum Gene Ther 25: 3\u201311","journal-title":"Hum Gene Ther"},{"key":"BFEMMM201506039_CR7","doi-asserted-by":"crossref","first-page":"488","DOI":"10.1161\/CIRCRESAHA.115.304351","volume":"115","author":"Q Ding","year":"2014","unstructured":"Ding Q, Strong A, Patel KM, Ng SL, Gosis BS, Regan SN, Cowan CA, Rader DJ, Musunuru K (2014) Permanent alteration of PCSK9 with in\u00a0vivo CRISPR\u2010Cas9 genome editing. Circ Res 115: 488\u2013492","journal-title":"Circ Res"},{"key":"BFEMMM201506039_CR8","doi-asserted-by":"crossref","first-page":"1258096","DOI":"10.1126\/science.1258096","volume":"346","author":"JA Doudna","year":"2014","unstructured":"Doudna JA, Charpentier E (2014) Genome editing. The new frontier of genome engineering with CRISPR\u2010Cas9. Science 346: 1258096","journal-title":"Science"},{"key":"BFEMMM201506039_CR9","doi-asserted-by":"crossref","first-page":"1051","DOI":"10.1038\/nmeth.3075","volume":"11","author":"M Holkers","year":"2014","unstructured":"Holkers M, Maggio I, Henriques SF, Janssen JM, Cathomen T, Goncalves MA (2014) Adenoviral vector DNA for accurate genome editing with engineered nucleases. Nat Methods 11: 1051\u20131057","journal-title":"Nat Methods"},{"key":"BFEMMM201506039_CR10","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1038\/nbt.2501","volume":"31","author":"WY Hwang","year":"2013","unstructured":"Hwang WY, Fu Y, Reyon D, Maeder ML, Tsai SQ, Sander JD, Peterson RT, Yeh JR, Joung JK (2013) Efficient genome editing in zebrafish using a CRISPR\u2010Cas system. Nat Biotechnol 31: 227\u2013229","journal-title":"Nat Biotechnol"},{"key":"BFEMMM201506039_CR11","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","volume":"337","author":"M Jinek","year":"2012","unstructured":"Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E (2012) A programmable dual\u2010RNA\u2010guided DNA endonuclease in adaptive bacterial immunity. Science 337: 816\u2013821","journal-title":"Science"},{"key":"BFEMMM201506039_CR12","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1126\/science.8211118","volume":"262","author":"MA Kay","year":"1993","unstructured":"Kay MA, Rothenberg S, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM et\u00a0al (1993) In vivo gene therapy of hemophilia B: sustained partial correction in factor IX\u2010deficient dogs. Science 262: 117\u2013119","journal-title":"Science"},{"key":"BFEMMM201506039_CR13","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1038\/73464","volume":"24","author":"MA Kay","year":"2000","unstructured":"Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW et\u00a0al (2000) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24: 257\u2013261","journal-title":"Nat Genet"},{"key":"BFEMMM201506039_CR14","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1038\/nrg2971","volume":"12","author":"MA Kay","year":"2011","unstructured":"Kay MA (2011) State\u2010of\u2010the\u2010art gene\u2010based therapies: the road ahead. Nat Rev Genet 12: 316\u2013328","journal-title":"Nat Rev Genet"},{"key":"BFEMMM201506039_CR15","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1038\/sj.cgt.7701119","volume":"15","author":"SE Khorsandi","year":"2008","unstructured":"Khorsandi SE, Bachellier P, Weber JC, Greget M, Jaeck D, Zacharoulis D, Rountas C, Helmy S, Helmy A, Al\u2010Waracky M et\u00a0al (2008) Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human. Cancer Gene Ther 15: 225\u2013230","journal-title":"Cancer Gene Ther"},{"key":"BFEMMM201506039_CR16","doi-asserted-by":"crossref","first-page":"784","DOI":"10.1182\/blood.V91.3.784","volume":"91","author":"SH Kung","year":"1998","unstructured":"Kung SH, Hagstrom JN, Cass D, Tai SJ, Lin HF, Stafford DW, High KA (1998) Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood 91: 784\u2013790","journal-title":"Blood"},{"key":"BFEMMM201506039_CR17","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nature10177","volume":"475","author":"H Li","year":"2011","unstructured":"Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L et\u00a0al (2011) In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475: 217\u2013221","journal-title":"Nature"},{"key":"BFEMMM201506039_CR18","doi-asserted-by":"crossref","first-page":"681","DOI":"10.1038\/nbt.2661","volume":"31","author":"D Li","year":"2013","unstructured":"Li D, Qiu Z, Shao Y, Chen Y, Guan Y, Liu M, Li Y, Gao N, Wang L, Lu X et\u00a0al (2013) Heritable gene targeting in the mouse and rat using a CRISPR\u2010Cas system. Nat Biotechnol 31: 681\u2013683","journal-title":"Nat Biotechnol"},{"key":"BFEMMM201506039_CR19","doi-asserted-by":"crossref","first-page":"e04766","DOI":"10.7554\/eLife.04766","volume":"3","author":"S Lin","year":"2014","unstructured":"Lin S, Staahl BT, Alla RK, Doudna JA (2014) Enhanced homology\u2010directed human genome engineering by controlled timing of CRISPR\/Cas9 delivery. Elife 3: e04766","journal-title":"Elife"},{"key":"BFEMMM201506039_CR20","doi-asserted-by":"crossref","first-page":"1258","DOI":"10.1038\/sj.gt.3300947","volume":"6","author":"F Liu","year":"1999","unstructured":"Liu F, Song Y, Liu D (1999) Hydrodynamics\u2010based transfection in animals by systemic administration of plasmid DNA. Gene Ther 6: 1258\u20131266","journal-title":"Gene Ther"},{"key":"BFEMMM201506039_CR21","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1126\/science.1254445","volume":"345","author":"C Long","year":"2014","unstructured":"Long C, McAnally JR, Shelton JM, Mireault AA, Bassel\u2010Duby R, Olson EN (2014) Prevention of muscular dystrophy in mice by CRISPR\/Cas9\u2010mediated editing of germline DNA. Science 345: 1184\u20131188","journal-title":"Science"},{"key":"BFEMMM201506039_CR22","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1126\/science.1232033","volume":"339","author":"P Mali","year":"2013","unstructured":"Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, Norville JE, Church GM (2013) RNA\u2010guided human genome engineering via Cas9. Science 339: 823\u2013826","journal-title":"Science"},{"key":"BFEMMM201506039_CR23","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1089\/10430349950018364","volume":"10","author":"DA Muruve","year":"1999","unstructured":"Muruve DA, Barnes MJ, Stillman IE, Libermann TA (1999) Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil\u2010dependent hepatic injury in\u00a0vivo. Hum Gene Ther 10: 965\u2013976","journal-title":"Hum Gene Ther"},{"key":"BFEMMM201506039_CR24","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","volume":"371","author":"AC Nathwani","year":"2014","unstructured":"Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D et\u00a0al (2014) Long\u2010term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 371: 1994\u20132004","journal-title":"N Engl J Med"},{"key":"BFEMMM201506039_CR25","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1016\/j.cell.2014.01.027","volume":"156","author":"Y Niu","year":"2014","unstructured":"Niu Y, Shen B, Cui Y, Chen Y, Wang J, Wang L, Kang Y, Zhao X, Si W, Li W et\u00a0al (2014) Generation of gene\u2010modified cynomolgus monkey via Cas9\/RNA\u2010mediated gene targeting in one\u2010cell embryos. Cell 156: 836\u2013843","journal-title":"Cell"},{"key":"BFEMMM201506039_CR26","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.stem.2015.07.001","volume":"17","author":"CY Park","year":"2015","unstructured":"Park CY, Kim DH, Son JS, Sung JJ, Lee J, Bae S, Kim JH, Kim DW, Kim JS (2015) Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient\u2010derived iPSCs using CRISPR\u2010Cas9. Cell Stem Cell 17: 213\u2013220","journal-title":"Cell Stem Cell"},{"key":"BFEMMM201506039_CR27","doi-asserted-by":"crossref","first-page":"1329","DOI":"10.1111\/jth.12276","volume":"11","author":"PM Rallapalli","year":"2013","unstructured":"Rallapalli PM, Kemball\u2010Cook G, Tuddenham EG, Gomez K, Perkins SJ (2013) An interactive mutation database for human coagulation factor IX provides novel insights into the phenotypes and genetics of hemophilia B. J Thromb Haemost 11: 1329\u20131340","journal-title":"J Thromb Haemost"},{"key":"BFEMMM201506039_CR28","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1038\/nature14299","volume":"520","author":"FA Ran","year":"2015","unstructured":"Ran FA, Cong L, Yan WX, Scott DA, Gootenberg JS, Kriz AJ, Zetsche B, Shalem O, Wu X, Makarova KS et\u00a0al (2015) In vivo genome editing using Staphylococcus aureus Cas9. Nature 520: 186\u2013191","journal-title":"Nature"},{"key":"BFEMMM201506039_CR29","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1016\/j.stem.2013.11.002","volume":"13","author":"G Schwank","year":"2013","unstructured":"Schwank G, Koo BK, Sasselli V, Dekkers JF, Heo I, Demircan T, Sasaki N, Boymans S, Cuppen E, van der Ent CK et\u00a0al (2013) Functional repair of CFTR by CRISPR\/Cas9 in intestinal stem cell organoids of cystic fibrosis patients. Cell Stem Cell 13: 653\u2013658","journal-title":"Cell Stem Cell"},{"key":"BFEMMM201506039_CR30","doi-asserted-by":"crossref","first-page":"2493","DOI":"10.1038\/nprot.2014.171","volume":"9","author":"Y Shao","year":"2014","unstructured":"Shao Y, Guan Y, Wang L, Qiu Z, Liu M, Chen Y, Wu L, Li Y, Ma X, Liu M et\u00a0al (2014) CRISPR\/Cas\u2010mediated genome editing in the rat via direct injection of one\u2010cell embryos. Nat Protoc 9: 2493\u20132512","journal-title":"Nat Protoc"},{"key":"BFEMMM201506039_CR31","doi-asserted-by":"crossref","first-page":"720","DOI":"10.1038\/cr.2013.46","volume":"23","author":"B Shen","year":"2013","unstructured":"Shen B, Zhang J, Wu H, Wang J, Ma K, Li Z, Zhang X, Zhang P, Huang X (2013) Generation of gene\u2010modified mice via Cas9\/RNA\u2010mediated gene targeting. Cell Res 23: 720\u2013723","journal-title":"Cell Res"},{"key":"BFEMMM201506039_CR32","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1038\/ng0797-270","volume":"16","author":"RO Snyder","year":"1997","unstructured":"Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK et\u00a0al (1997) Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 16: 270\u2013276","journal-title":"Nat Genet"},{"key":"BFEMMM201506039_CR33","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/0076-6879(93)22011-4","volume":"222","author":"AR Thompson","year":"1993","unstructured":"Thompson AR, Chen SH (1993) Characterization of factor IX defects in hemophilia B patients. Methods Enzymol 222: 143\u2013169","journal-title":"Methods Enzymol"},{"key":"BFEMMM201506039_CR34","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1016\/j.cell.2013.04.025","volume":"153","author":"H Wang","year":"2013","unstructured":"Wang H, Yang H, Shivalila CS, Dawlaty MM, Cheng AW, Zhang F, Jaenisch R (2013) One\u2010step generation of mice carrying mutations in multiple genes by CRISPR\/Cas\u2010mediated genome engineering. Cell 153: 910\u2013918","journal-title":"Cell"},{"key":"BFEMMM201506039_CR35","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1055\/s-0037-1615621","volume":"85","author":"GC White 2nd","year":"2001","unstructured":"White GC 2nd, Rosendaal F, Aledort LM, Lusher JM, Rothschild C, Ingerslev J, Factor V, Factor IXS (2001) Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost 85: 560","journal-title":"Thromb Haemost"},{"key":"BFEMMM201506039_CR36","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1016\/j.stem.2013.10.016","volume":"13","author":"Y Wu","year":"2013","unstructured":"Wu Y, Liang D, Wang Y, Bai M, Tang W, Bao S, Yan Z, Li D, Li J (2013) Correction of a genetic disease in mouse via use of CRISPR\u2010Cas9. Cell Stem Cell 13: 659\u2013662","journal-title":"Cell Stem Cell"},{"key":"BFEMMM201506039_CR37","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1038\/75568","volume":"25","author":"SR Yant","year":"2000","unstructured":"Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA (2000) Somatic integration and long\u2010term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet 25: 35\u201341","journal-title":"Nat Genet"},{"key":"BFEMMM201506039_CR38","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/nbt.2884","volume":"32","author":"H Yin","year":"2014","unstructured":"Yin H, Xue W, Chen S, Bogorad RL, Benedetti E, Grompe M, Koteliansky V, Sharp PA, Jacks T, Anderson DG (2014) Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 32: 551\u2013553","journal-title":"Nat Biotechnol"}],"container-title":["EMBO Molecular Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.15252\/emmm.201506039.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.15252\/emmm.201506039","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.15252\/emmm.201506039\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.embopress.org\/doi\/pdf\/10.15252\/emmm.201506039","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.15252\/emmm.201506039.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,18]],"date-time":"2026-05-18T06:14:03Z","timestamp":1779084843000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.15252\/emmm.201506039"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,3,10]]},"references-count":38,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2016,5,1]]}},"alternative-id":["BFEMMM201506039"],"URL":"https:\/\/doi.org\/10.15252\/emmm.201506039","relation":{},"ISSN":["1757-4676","1757-4684"],"issn-type":[{"value":"1757-4676","type":"print"},{"value":"1757-4684","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,3,10]]},"assertion":[{"value":"8 November 2015","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"17 February 2016","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"18 February 2016","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"10 March 2016","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors declare that they have no conflict of interest.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}