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      KCI등재 SCIE SCOPUS

      Humanized model mice by genome editing and engraftment technologies

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      https://www.riss.kr/link?id=A106056269

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      다국어 초록 (Multilingual Abstract)

      Purpose of review: In drug development, non-clinical studies are performed to evaluate the feasibility, iterative testing and safety of a drug. To harness this process, small animal models which are inexpensive, and easy to breed and maintain such as mice and rats are preferred for non-clinical studies. However, humans and these animals share a large portion of genetic makeup, but genetic and physiological gaps are unavoidable. Efforts to address this innate difference between humans and animals have been made by establishing a so called ‘humanized’ mouse. In this review, we summarize the scope of the ‘humanization’ with genome editing technology as well as with cell/ tissue engraftment.
      Recent findings: A specifically targeted genetic manipulation became feasible by the development genome editing technologies including zinc finger nucleases (ZFN), transcription activator-like effector nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. A humanized animal model can also be generated by engraft human cells or tissues into the corresponding sites of animals. If these two approaches are combined in a synergistic manner, a ‘humanized mouse’ would be better used for non-clinical study in various experimental and clinical realms.
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      Purpose of review: In drug development, non-clinical studies are performed to evaluate the feasibility, iterative testing and safety of a drug. To harness this process, small animal models which are inexpensive, and easy to breed and maintain such as ...

      Purpose of review: In drug development, non-clinical studies are performed to evaluate the feasibility, iterative testing and safety of a drug. To harness this process, small animal models which are inexpensive, and easy to breed and maintain such as mice and rats are preferred for non-clinical studies. However, humans and these animals share a large portion of genetic makeup, but genetic and physiological gaps are unavoidable. Efforts to address this innate difference between humans and animals have been made by establishing a so called ‘humanized’ mouse. In this review, we summarize the scope of the ‘humanization’ with genome editing technology as well as with cell/ tissue engraftment.
      Recent findings: A specifically targeted genetic manipulation became feasible by the development genome editing technologies including zinc finger nucleases (ZFN), transcription activator-like effector nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. A humanized animal model can also be generated by engraft human cells or tissues into the corresponding sites of animals. If these two approaches are combined in a synergistic manner, a ‘humanized mouse’ would be better used for non-clinical study in various experimental and clinical realms.

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      참고문헌 (Reference)

      1 Gaj, T., "ZFN, TALEN and CRISPR/Cas-based methods for genome engineering" 31 : 397-405, 2013

      2 Liao, H. K., "Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells" 6 : 6413-, 2015

      3 Hornick, R. B., "Typhoid fever: pathogenesis and immunologic control" 283 : 686-691, 1970

      4 Wain, J., "Typhoid fever" 385 : 1136-1145, 2015

      5 Hunter, P., "The paradox of model organisms. The use of model organisms in research will continue despite their shortcomings" 9 : 717-720, 2008

      6 Vercauteren, K., "Targeting a host-cell entry factor barricades antiviral-resistant HCV variants from on-therapy breakthrough in human-liver mice" 65 : 2029-2034, 2016

      7 Lloyd, A., "Targeted mutagenesis using zinc-finger nucleases in Arabidopsis" 102 : 2232-2237, 2005

      8 Cui, X., "Targeted integration in rat and mouse embryos with zinc-finger nucleases" 29 : 64-67, 2011

      9 Carbery, I. D., "Targeted genome modification in mice using zinc-finger nucleases" 186 : 451-459, 2010

      10 Greek, R., "Systematic reviews of animal models: methodology versus epistemology" 10 : 206-221, 2013

      1 Gaj, T., "ZFN, TALEN and CRISPR/Cas-based methods for genome engineering" 31 : 397-405, 2013

      2 Liao, H. K., "Use of the CRISPR/Cas9 system as an intracellular defense against HIV-1 infection in human cells" 6 : 6413-, 2015

      3 Hornick, R. B., "Typhoid fever: pathogenesis and immunologic control" 283 : 686-691, 1970

      4 Wain, J., "Typhoid fever" 385 : 1136-1145, 2015

      5 Hunter, P., "The paradox of model organisms. The use of model organisms in research will continue despite their shortcomings" 9 : 717-720, 2008

      6 Vercauteren, K., "Targeting a host-cell entry factor barricades antiviral-resistant HCV variants from on-therapy breakthrough in human-liver mice" 65 : 2029-2034, 2016

      7 Lloyd, A., "Targeted mutagenesis using zinc-finger nucleases in Arabidopsis" 102 : 2232-2237, 2005

      8 Cui, X., "Targeted integration in rat and mouse embryos with zinc-finger nucleases" 29 : 64-67, 2011

      9 Carbery, I. D., "Targeted genome modification in mice using zinc-finger nucleases" 186 : 451-459, 2010

      10 Greek, R., "Systematic reviews of animal models: methodology versus epistemology" 10 : 206-221, 2013

      11 Mizuno, S., "Simple generation of albino C57BL/6 J mice with G291 T mutation in the tyrosinase gene by the CRISPR/Cas9 system" 25 : 327-334, 2014

      12 Dougan, G., "Salmonella enterica serovar Typhi and the pathogenesis of typhoid fever" 68 : 317-336, 2014

      13 Heyer, W. D., "Regulation of homologous recombination in eukaryotes" 44 : 113-139, 2010

      14 Ishikawa, F., "Purified human hematopoietic stem cells contribute to the generation of cardiomyocytes through cell fusion" 20 : 950-952, 2006

      15 Yang, H., "One-step generation of mice carrying reporter and conditional alleles by CRISPR/Cas-mediated genome engineering" 154 : 1370-1379, 2013

      16 Wang, H., "One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering" 154 : 1370-1379, 2013

      17 Pearson, T., "Non-obese diabetic recombination activating gene-1 ($NOD-Rag1^{null}$) interleukin (IL)-2 receptor common gamma chain ($IL2{\gamma}^{null}$) null mice: a radioresistant model for human lymphohaematopoietic engraftment" 154 : 270-284, 2008

      18 Ito, M., "NOD/SCID/cnul mouse: an excellent recipiet mouse model for engraftment of human cells" 100 : 3175-3182, 2002

      19 Shultz, L. D., "NOD/LtSz-Rag1nullPfpnull mice a new model system with increased levels of human peripheral leukocyte and hematopoietic stem-cell engraftment" 76 : 1036-1042, 2003

      20 Shultz, L. D., "Multiple defects in innate and adaptive immunologic function in NOD/LtSz-scid mice" 154 : 180-191, 1995

      21 Bility, M. T., "Liver immunepathogenesis and therapy of human liver tropic virus infection in humanized mouse models" 28 : 120-124, 2013

      22 Samraj, A. N., "Involvement of a non-human sialic Acid in human cancer" 4 : 33-, 2014

      23 Yang, Y., "In vivo tropism of Salmonella Typhi toxin to cells expressing a multiantennal glycan receptor" 3 : 155-163, 2018

      24 Libby, S. J., "Humanized nonobese diabetic-scid IL2rgammanull mice are susceptible to lethal Salmonella typhi infection" 107 : 15589-15594, 2010

      25 Shultz, L. D., "Humanized mice in translational biomedical research" 7 : 118-130, 2007

      26 Shultz, L. D., "Humanized mice for immune system investigation: progress, promise and challenges" 12 : 786-798, 2012

      27 Mian, M. F., "Humanized mice for Salmonella typhi infection: new tools for an old problem" 23 : 248-252, 2011

      28 Firoz Mian, M., "Humanized mice are susceptible to Salmonella typhi infection" 8 : 83-87, 2011

      29 Hu, Y., "Human pharmacogenetics analysis in chimeric mice with 'humanized livers'" 23 : 78-83, 2013

      30 Mercer, D. F., "Hepatitis C virus replication in mice with chimeric human livers" 7 : 927-933, 2001

      31 Bility, M. T., "Hepatitis B virus infection and immune-pathogenesis in a humanized mouse model: induction of human-specific liver fibrosis and M2-like macrophages" 10 : e1004032-, 2014

      32 Hockemeyer, D., "Genetic engineering of human pluripotent cells using TALE nucleases" 29 : 731-734, 2011

      33 Schumann, K., "Generation of knock-in primary human T cells using Cas9 ribonucleoproteins" 112 : 10437-10442, 2015

      34 Porteus, M. H., "Gene targeting using zinc finger nucleases" 23 : 967-973, 2005

      35 Suemizu, H., "Establishment of a humanized model of liver using NOD/Shi-scid IL2Rgnull mice" 377 : 248-252, 2008

      36 Crump, J. A., "Epidemiology, clinical presentation, laboratory diagnosis, antimicrobial resistance, and antimicrobial management of invasive salmonella infections" 28 : 901-937, 2015

      37 Christianson, S. W., "Enhanced human CD4+ T cell engraftment in β2-microglobulin-deficient NODscid mice" 158 : 3578-3586, 1997

      38 Traggiai, E., "Development of a human adaptive immune system in cord blood cell-transplanted mice" 304 : 104-107, 2004

      39 Hiramatsu, H., "Complete reconstitution of human lymphocytes from cord blood CD34+ cells using the NOD/SCID/cnul mice model" 102 : 873-880, 2003

      40 de Jong, Y. P., "Broadly neutralizing antibodies abrogate established hepatitis C virus infection" 6 : 254ra129-, 2014

      41 Boch, J., "Breaking the code of DNA binding specificity of TAL-type III effectors" 326 : 1509-1512, 2009

      42 Tran Vu Thieu, N., "An evaluation of purified Salmonella Typhi protein antigens for the serological diagnosis of acute typhoid fever" 75 : 104-114, 2017

      43 Mural, R. J., "Acomparison of whole-genome shotgun-derived mouse chromosome 16 and the human genome" 296 : 1661-1671, 2002

      44 Moscou, M. J., "A simple cipher governs DNA recognition by TAL effectors" 326 : 1501-, 2009

      45 Bosma, G. C., "A severe combined immunodeficiency mutation in the mouse" 301 : 527-530, 1983

      46 Jinek, M., "A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity" 337 : 816-821, 2012

      47 Song, J., "A mouse model for the human pathogen Salmonella typhi" 8 : 369-376, 2010

      48 Washburn, M. L., "A humanized mouse model to study hepatitis C virus infection, immuneresponse, and liver disease" 140 : 1334-1344, 2011

      49 Robinet, E., "A first step towards a mouse model for hepatitis C virus infection containing a human immune system" 55 : 718-720, 2011

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      2021-01-01 평가 등재학술지 선정 (해외등재 학술지 평가) KCI등재
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      2013-10-01 평가 등재학술지 선정 (기타) KCI등재
      2011-01-01 평가 등재후보학술지 유지 (기타) KCI등재후보
      2007-01-01 평가 SCIE 등재 (신규평가) KCI등재후보
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