메뉴 건너뛰기




Volumn 34, Issue 1, 2012, Pages 1-13

Induced pluripotent stem cells in research and therapy

Author keywords

Disease modelling; Embryonic like properties; Gene therapy; Patient specific therapy; Regenerative medicine

Indexed keywords

KRUPPEL LIKE FACTOR 4; LENTIVIRUS VECTOR; LIN 28 PROTEIN; MICRORNA; MICRORNA 200C; MICRORNA 302S; MICRORNA 369S; MYC PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; PROTEIN; RETROVIRUS VECTOR; TRANSCRIPTION FACTOR; TRANSCRIPTION FACTOR NANOG; TRANSCRIPTION FACTOR SOX2; UNCLASSIFIED DRUG; VALPROIC ACID;

EID: 84863310681     PISSN: 01268635     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review
Times cited : (20)

References (96)
  • 1
    • 36248966518 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from adult human fibroblasts by defined factors
    • Takahashi K, Tanabe K, Ohnuki M, et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007;131:861-72.
    • (2007) Cell , vol.131 , pp. 861-872
    • Takahashi, K.1    Tanabe, K.2    Ohnuki, M.3
  • 2
    • 36749043230 scopus 로고    scopus 로고
    • Induced pluripotent stem cell lines derived from human somatic cells
    • Yu J, Vodyanik MA, Smuga-Otto K, et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007;318:1917-20.
    • (2007) Science , vol.318 , pp. 1917-1920
    • Yu, J.1    Vodyanik, M.A.2    Smuga-Otto, K.3
  • 3
    • 0000062301 scopus 로고
    • Cytological demonstration of the clonal nature of spleen colonies derived from transplanted mouse marrow cells
    • Becker AJ, McCulloch EA, Till JE. Cytological demonstration of the clonal nature of spleen colonies derived from transplanted mouse marrow cells. Nature 1963;197: 452-4.
    • (1963) Nature , vol.197 , pp. 452-454
    • Becker, A.J.1    McCulloch, E.A.2    Till, J.E.3
  • 4
    • 27944478942 scopus 로고    scopus 로고
    • Embryonic stem cells: Understanding their history, cell biology and signalling
    • Friel R, van der Sar S, Mee PJ. Embryonic stem cells: understanding their history, cell biology and signalling. Adv Drug Deliv Rev 2005;57:1894-903.
    • (2005) Adv Drug Deliv Rev , vol.57 , pp. 1894-1903
    • Friel, R.1    van der Sar, S.2    Mee, P.J.3
  • 5
    • 0032491416 scopus 로고    scopus 로고
    • Embryonic stem cell lines derived from human blastocysts
    • Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. Embryonic stem cell lines derived from human blastocysts. Science 1998;282:1145-7.
    • (1998) Science , vol.282 , pp. 1145-1147
    • Thomson, J.A.1    Itskovitz-Eldor, J.2    Shapiro, S.S.3
  • 7
    • 18644384444 scopus 로고    scopus 로고
    • Human embryonic stem cell-derived oligodendrocyteprogenitor cell transplants remyelinate and restore locomotion after spinal cord injury
    • Keirstead HS, Nistor G, Bernal G, et al. Human embryonic stem cell-derived oligodendrocyteprogenitor cell transplants remyelinate and restore locomotion after spinal cord injury. J Neurosci 2005;25:4694-705.
    • (2005) J Neurosci , vol.25 , pp. 4694-4705
    • Keirstead, H.S.1    Nistor, G.2    Bernal, G.3
  • 8
    • 50149098605 scopus 로고    scopus 로고
    • Induced pluripotent stem cells generated from patients fromALS can be differentiated into motor neurons
    • Dimos JT, Rodolfa KT, Niakan KK, et al. Induced pluripotent stem cells generated from patients fromALS can be differentiated into motor neurons. Science 2008;321:1218-21.
    • (2008) Science , vol.321 , pp. 1218-1221
    • Dimos, J.T.1    Rodolfa, K.T.2    Niakan, K.K.3
  • 9
    • 38349004470 scopus 로고    scopus 로고
    • Multiple layers of molecular controls modulate self-renewal and neuronal lineage specification of embryonic stem cells
    • Yang D, Zhang ZJ, Oldenburg M, et al. Multiple layers of molecular controls modulate self-renewal and neuronal lineage specification of embryonic stem cells. Stem Cells 2008; 26:55-63.
    • (2008) Stem Cells , vol.26 , pp. 55-63
    • Yang, D.1    Zhang, Z.J.2    Oldenburg, M.3
  • 10
    • 0036816302 scopus 로고    scopus 로고
    • Human embryonic stem cell lines: Socio-legal concerns and therapeutic promise
    • McLaren A. Human embryonic stem cell lines: socio-legal concerns and therapeutic promise. C R Biologies 2002;325:1009-12.
    • (2002) C R Biologies , vol.325 , pp. 1009-1012
    • McLaren, A.1
  • 11
    • 33747195353 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors
    • Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006;126:663-76.
    • (2006) Cell , vol.126 , pp. 663-676
    • Takahashi, K.1    Yamanaka, S.2
  • 12
    • 55849115999 scopus 로고    scopus 로고
    • Induced pluripotent stem cells generated without viral integration
    • Stadfeld M, Nagaya M, Utikal J, et al. Induced pluripotent stem cells generated without viral integration. Science 2008;322:945-9.
    • (2008) Science , vol.322 , pp. 945-949
    • Stadfeld, M.1    Nagaya, M.2    Utikal, J.3
  • 13
    • 55849117368 scopus 로고    scopus 로고
    • Generation of mouse induced pluripotent stem cells without viral vectors
    • Okita K, Nakagawa M, Hyenjong H, et al. Generation of mouse induced pluripotent stem cells without viral vectors. Science 2008;322:949-53.
    • (2008) Science , vol.322 , pp. 949-953
    • Okita, K.1    Nakagawa, M.2    Hyenjong, H.3
  • 14
    • 64749111225 scopus 로고    scopus 로고
    • Virus-free induction of pluripotency and subsequent excision of reprogramming factors
    • Kaji K, Norrby K, Paca A, et al. Virus-free induction of pluripotency and subsequent excision of reprogramming factors. Nature 2009;458:771-5.
    • (2009) Nature , vol.458 , pp. 771-775
    • Kaji, K.1    Norrby, K.2    Paca, A.3
  • 15
    • 66049135249 scopus 로고    scopus 로고
    • Generation of induced pluripotent stem cells using recombinant proteins
    • Zhou H, Wu S, Joo JY, et al. Generation of induced pluripotent stem cells using recombinant proteins. Cell Stem Cell 2009;4:381-4.
    • (2009) Cell Stem Cell , vol.4 , pp. 381-384
    • Zhou, H.1    Wu, S.2    Joo, J.Y.3
  • 16
    • 66049143859 scopus 로고    scopus 로고
    • Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins
    • Kim D, Kim CH, Moon JI, et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell 2009;4:472-6.
    • (2009) Cell Stem Cell , vol.4 , pp. 472-476
    • Kim, D.1    Kim, C.H.2    Moon, J.I.3
  • 17
    • 55749089875 scopus 로고    scopus 로고
    • Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes
    • Aasen T, Raya A, Barrero MJ, et al. Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes. Nat Biotechnol 2008;26:1276-84.
    • (2008) Nat Biotechnol , vol.26 , pp. 1276-1284
    • Aasen, T.1    Raya, A.2    Barrero, M.J.3
  • 18
    • 67049164945 scopus 로고    scopus 로고
    • Generation of induced pluripotent stem cells from human blood
    • Loh YH, Agarwal S, Park IH, et al. Generation of induced pluripotent stem cells from human blood. Blood 2009;113:5476-9.
    • (2009) Blood , vol.113 , pp. 5476-5479
    • Loh, Y.H.1    Agarwal, S.2    Park, I.H.3
  • 19
    • 41349090105 scopus 로고    scopus 로고
    • Eneration of human induced pluripotent stem cells from dermal fibroblasts
    • Lowry WE, Richter L, Yachechko R, et al. eneration of human induced pluripotent stem cells from dermal fibroblasts. Proc Natl Acad Sci USA 2008;105:2883-8.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 2883-2888
    • Lowry, W.E.1    Richter, L.2    Yachechko, R.3
  • 20
    • 55749104227 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from primary human fibroblasts with only Oct4 and Sox2
    • Huangfu D, Osafune K, Maehr R, et al. Induction of pluripotent stem cells from primary human fibroblasts with only Oct4 and Sox2. Nat Biotechnol 2008;26:1269-75.
    • (2008) Nat Biotechnol , vol.26 , pp. 1269-1275
    • Huangfu, D.1    Osafune, K.2    Maehr, R.3
  • 21
    • 45449094570 scopus 로고    scopus 로고
    • Reprogramming of pancreatic β cells into induced pluripotent stem cells
    • Stadtfeld M, Brennand K, Hochedlinger K. Reprogramming of pancreatic β cells into induced pluripotent stem cells. Curr Biol 2008;18:890-4.
    • (2008) Curr Biol , vol.18 , pp. 890-894
    • Stadtfeld, M.1    Brennand, K.2    Hochedlinger, K.3
  • 22
    • 77958082207 scopus 로고    scopus 로고
    • Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette
    • Somers A, Jean JC, Sommer CA, et al. Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells 2010;28:1728-40.
    • (2010) Stem Cells , vol.28 , pp. 1728-1740
    • Somers, A.1    Jean, J.C.2    Sommer, C.A.3
  • 23
    • 79953881831 scopus 로고    scopus 로고
    • Highly efficient miRNA-mediated reprogramming of mouse and human somatic cells to pluripotency
    • Anokye-Danso F, Trivedi CM, Juhr D, et al. Highly efficient miRNA-mediated reprogramming of mouse and human somatic cells to pluripotency. Cell Stem Cell 2011;8;376-88.
    • (2011) Cell Stem Cell , vol.8 , pp. 376-388
    • Anokye-Danso, F.1    Trivedi, C.M.2    Juhr, D.3
  • 24
    • 50849142559 scopus 로고    scopus 로고
    • A high-efficiency system for the generation and study of human induced pluripotent stem cells
    • Maherali N, Ahfeldt T, Rigamonti A, et al. A high-efficiency system for the generation and study of human induced pluripotent stem cells. Cell Stem Cell 2008;3:340-5.
    • (2008) Cell Stem Cell , vol.3 , pp. 340-345
    • Maherali, N.1    Ahfeldt, T.2    Rigamonti, A.3
  • 25
    • 39149115929 scopus 로고    scopus 로고
    • Defining molecular cornerstones during fibroblast to iPS cell reprogramming in mouse
    • Stadtfeld M, Maherali N, Breault DT, et al. Defining molecular cornerstones during fibroblast to iPS cell reprogramming in mouse. Cell Stem Cell 2008;2:230-40.
    • (2008) Cell Stem Cell , vol.2 , pp. 230-240
    • Stadtfeld, M.1    Maherali, N.2    Breault, D.T.3
  • 26
    • 77955118962 scopus 로고    scopus 로고
    • Generation of human induced pluripotent stem cells by simple transient transfection of plasmid DNA encoding reprogramming factors
    • Si-Tayeb K, Noto FK, Sepac A, et al. Generation of human induced pluripotent stem cells by simple transient transfection of plasmid DNA encoding reprogramming factors. BMC Dev Biol 2010;10:81.
    • (2010) BMC Dev Biol , vol.10 , pp. 81
    • Si-Tayeb, K.1    Noto, F.K.2    Sepac, A.3
  • 27
    • 64749083939 scopus 로고    scopus 로고
    • Piggyback transposition reprograms fibroblasts to induced pluripotent stem cells
    • Woltjen K, Michael IP, Mohseni P, et al. piggyback transposition reprograms fibroblasts to induced pluripotent stem cells. Nature 2009;458:766-70.
    • (2009) Nature , vol.458 , pp. 766-770
    • Woltjen, K.1    Michael, I.P.2    Mohseni, P.3
  • 28
    • 70450265981 scopus 로고    scopus 로고
    • Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome
    • Fusaki N, Ban H, Nishiyama A, et al. Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome. Proc Jpn Acad 2009;85:348-62.
    • (2009) Proc Jpn Acad , vol.85 , pp. 348-362
    • Fusaki, N.1    Ban, H.2    Nishiyama, A.3
  • 29
    • 77958536106 scopus 로고    scopus 로고
    • Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA
    • Warren L, Manos PD, Ahfeldt T, et al. Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA. Cell Stem Cell 2010;7:618-30.
    • (2010) Cell Stem Cell , vol.7 , pp. 618-630
    • Warren, L.1    Manos, P.D.2    Ahfeldt, T.3
  • 30
    • 79957855262 scopus 로고    scopus 로고
    • Reprogramming of mouse and human cells to pluripotency using mature microRNAs
    • Miyoshi N, Ishii H, Nagano H, et al. Reprogramming of mouse and human cells to pluripotency using mature microRNAs. Cell Stem Cell 2011;8: 633-8.
    • (2011) Cell Stem Cell , vol.8 , pp. 633-638
    • Miyoshi, N.1    Ishii, H.2    Nagano, H.3
  • 31
    • 23944457397 scopus 로고    scopus 로고
    • Nuclear reprogramming of somatic cells after fusion with human embryonic stem cells
    • Cowan CA, Atienza J, Melton DA, et al. Nuclear reprogramming of somatic cells after fusion with human embryonic stem cells. Science 2005;309:1369-73.
    • (2005) Science , vol.309 , pp. 1369-1373
    • Cowan, C.A.1    Atienza, J.2    Melton, D.A.3
  • 32
    • 77957895936 scopus 로고    scopus 로고
    • A combined epigenetic and non-genetic approach for reprogramming human somatic cells
    • Han J, Sachdev PS, Sidhu KS. A combined epigenetic and non-genetic approach for reprogramming human somatic cells. PLoS One 2010;5(8): e12297.
    • (2010) PLoS One , vol.5 , Issue.8
    • Han, J.1    Sachdev, P.S.2    Sidhu, K.S.3
  • 33
    • 79957788311 scopus 로고    scopus 로고
    • Induced pluripotent stem cells for regenerative cardiovascular therapies and biomedical discovery
    • Nsair A, MacLellan WR. Induced pluripotent stem cells for regenerative cardiovascular therapies and biomedical discovery. Adv Drug Deliv Rev 2011;63:324-30.
    • (2011) Adv Drug Deliv Rev , vol.63 , pp. 324-330
    • Nsair, A.1    Maclellan, W.R.2
  • 34
    • 42649130264 scopus 로고    scopus 로고
    • Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease
    • Wernig M, Zhao JP, Pruszak J, et al. Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease. Proc Natl Acad Sci USA 2008;105:5856-61.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 5856-5861
    • Wernig, M.1    Zhao, J.P.2    Pruszak, J.3
  • 35
    • 84862777929 scopus 로고    scopus 로고
    • Clinical applications of patient-specific induced pluripotent stem cells in cardiovascular medicine
    • Oh YZ, Wei HM, Ma DR, et al. Clinical applications of patient-specific induced pluripotent stem cells in cardiovascular medicine. Heart 2012;98(6):443-9.
    • (2012) Heart , vol.98 , Issue.6 , pp. 443-449
    • Oh, Y.Z.1    Wei, H.M.2    Ma, D.R.3
  • 36
    • 84873337576 scopus 로고    scopus 로고
    • Characterization of human Huntington's disease cell model from induced pluripotent stem cells
    • Zhang N, An MC, Montoro D, et al. Characterization of human Huntington's disease cell model from induced pluripotent stem cells. PLoS Curr 2010;2:RRN1193.
    • (2010) PLoS Curr , vol.2
    • Zhang, N.1    An, M.C.2    Montoro, D.3
  • 37
    • 78650995671 scopus 로고    scopus 로고
    • A human iPSC model of Hutchinson Gilford progeria reveals vascular smooth muscle and mesenchymal stem cell defects
    • Zhang JQ, Lian QZ, Zhu GL, et al. A human iPSC model of Hutchinson Gilford progeria reveals vascular smooth muscle and mesenchymal stem cell defects. Cell Stem Cell 2011;8:31-45.
    • (2011) Cell Stem Cell , vol.8 , pp. 31-45
    • Zhang, J.Q.1    Lian, Q.Z.2    Zhu, G.L.3
  • 38
    • 79954626173 scopus 로고    scopus 로고
    • Recapitulationof premature ageing with iPSCs from Hutchinson-Gilford progeria syndrome
    • Liu GH, Barkho BZ, Ruiz S, et al. Recapitulationof premature ageing with iPSCs from Hutchinson-Gilford progeria syndrome. Nature 2011;472(7342):221-5.
    • (2011) Nature , vol.472 , Issue.7342 , pp. 221-225
    • Liu, G.H.1    Barkho, B.Z.2    Ruiz, S.3
  • 39
    • 77956214743 scopus 로고    scopus 로고
    • Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells
    • Urbach A, Bar-Nur O, Daley GQ, et al. Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells. Cell Stem Cell 2010;6(5):407-11.
    • (2010) Cell Stem Cell , vol.6 , Issue.5 , pp. 407-411
    • Urbach, A.1    Bar-Nur, O.2    Daley, G.Q.3
  • 40
    • 50549089957 scopus 로고    scopus 로고
    • Disease-specific induced pluripotent stem cells
    • Park IH, Arora N, Huo H, et al. Disease-specific induced pluripotent stem cells. Cell 2008;134(5):877-86.
    • (2008) Cell , vol.134 , Issue.5 , pp. 877-886
    • Park, I.H.1    Arora, N.2    Huo, H.3
  • 41
    • 77957729169 scopus 로고    scopus 로고
    • Patient-specific induced pluripotent stem-cell models for long-QT syndrome
    • Moretti A, Bellin M, Welling A, et al. Patient-specific induced pluripotent stem-cell models for long-QT syndrome. N Engl J Med 2010;363:1397e409.
    • (2010) N Engl J Med , vol.363
    • Moretti, A.1    Bellin, M.2    Welling, A.3
  • 42
    • 79954523845 scopus 로고    scopus 로고
    • Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation
    • Matsa E, Rajamohan D, Dick E, et al. Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation. Eur Heart J 2011;32:952e62.
    • (2011) Eur Heart J , vol.32
    • Matsa, E.1    Rajamohan, D.2    Dick, E.3
  • 43
    • 77953250105 scopus 로고    scopus 로고
    • Generation of iPSCs from cultured human malignant cells
    • Carette JE, Pruszak J, Varadarajan M, et al. Generation of iPSCs from cultured human malignant cells. Blood 2010;115:4039-42.
    • (2010) Blood , vol.115 , pp. 4039-4042
    • Carette, J.E.1    Pruszak, J.2    Varadarajan, M.3
  • 44
    • 76249133692 scopus 로고    scopus 로고
    • Defined factors induce reprogramming of gastrointestinal cancer cells
    • Miyoshi N, Ishii H, Nagai K, et al. Defined factors induce reprogramming of gastrointestinal cancer cells. Proc Natl Acad Sci USA 2010;107(1):40-45.
    • (2010) Proc Natl Acad Sci USA , vol.107 , Issue.1 , pp. 40-45
    • Miyoshi, N.1    Ishii, H.2    Nagai, K.3
  • 45
    • 84870063669 scopus 로고    scopus 로고
    • Acquired cancer stem cell phenotypes through Oct4-mediated dedifferentiation
    • Kumar SM, Liu S, Lu H, et al. Acquired cancer stem cell phenotypes through Oct4-mediated dedifferentiation. Oncogene 2012:1-14.
    • (2012) Oncogene , pp. 1-14
    • Kumar, S.M.1    Liu, S.2    Lu, H.3
  • 46
    • 79957868170 scopus 로고    scopus 로고
    • Induced pluripotent stem cells: A novel frontier in the study of human primary immunodeficiencies
    • Pessach IM, Ordovas-Montanes J, Zhang SY, et al. Induced pluripotent stem cells: a novel frontier in the study of human primary immunodeficiencies. J Allergy Clin Immunol 2011;127(6):1400-7.
    • (2011) J Allergy Clin Immunol , vol.127 , Issue.6 , pp. 1400-1407
    • Pessach, I.M.1    Ordovas-Montanes, J.2    Zhang, S.Y.3
  • 47
    • 4344645978 scopus 로고    scopus 로고
    • Can the pharmaceutical industry reduce attrition rates?
    • Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov 2004;3:711-6.
    • (2004) Nat Rev Drug Discov , vol.3 , pp. 711-716
    • Kola, I.1    Landis, J.2
  • 49
    • 78650469789 scopus 로고    scopus 로고
    • Applications of patient-specific induced pluripotent stem cells; focused on disease modeling, drug screening and therapeutic potentials for liver disease (review)
    • Chun YS, Chaudhari P, Jang YY. Applications of patient-specific induced pluripotent stem cells; focused on disease modeling, drug screening and therapeutic potentials for liver disease (review). Int J Biol Sci 2010;7:796-805.
    • (2010) Int J Biol Sci , vol.7 , pp. 796-805
    • Chun, Y.S.1    Chaudhari, P.2    Jang, Y.Y.3
  • 50
    • 77952250654 scopus 로고    scopus 로고
    • Human stem cell models for predictive cardiac safety pharmacology
    • Braam SR, Mummery CL. Human stem cell models for predictive cardiac safety pharmacology. Stem Cell Res 2010;4(3)155-6.
    • (2010) Stem Cell Res , vol.4 , Issue.3 , pp. 155-156
    • Braam, S.R.1    Mummery, C.L.2
  • 51
    • 77952253259 scopus 로고    scopus 로고
    • Progressive maturation in contracting cardiomyocytes derived from human embryonic stem cells: Qualitative effects on electrophysiological responses to drugs
    • Otsuji TG, Kurose Y, Yamauchi K, et al. Progressive maturation in contracting cardiomyocytes derived from human embryonic stem cells: qualitative effects on electrophysiological responses to drugs. Stem Cell Res 2010;4(3):201-13.
    • (2010) Stem Cell Res , vol.4 , Issue.3 , pp. 201-213
    • Otsuji, T.G.1    Kurose, Y.2    Yamauchi, K.3
  • 52
    • 70349301819 scopus 로고    scopus 로고
    • Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs
    • Lee G, Papapetrou EP, Kim H, et al. Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature 2009;461(7262):402-6.
    • (2009) Nature , vol.461 , Issue.7262 , pp. 402-406
    • Lee, G.1    Papapetrou, E.P.2    Kim, H.3
  • 53
    • 33645127443 scopus 로고    scopus 로고
    • Genetic medicines: Treatment strategies for hereditary disorders
    • O'Connor TP, Crystal RG. Genetic medicines: treatment strategies for hereditary disorders. Nat Rev Genet 2006;7:261-76.
    • (2006) Nat Rev Genet , vol.7 , pp. 261-276
    • O'Connor, T.P.1    Crystal, R.G.2
  • 54
    • 84888105678 scopus 로고    scopus 로고
    • [World Wide Web site]. Bethesda, MD: National Institutes of Health, U.S. Department of Health and Human Services, [cited Sunday, April 15, 2012] Available at
    • Zwaka TP. 2009. In Stem Cell Information [World Wide Web site]. Bethesda, MD: National Institutes of Health, U.S. Department of Health and Human Services, 2009 [cited Sunday, April 15, 2012] Available at http://stemcells.nih.gov/info/scireport/2006chapter4.
    • (2009) Stem Cell Information
    • Zwaka, T.P.1
  • 55
    • 0037448352 scopus 로고    scopus 로고
    • A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
    • Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-6.
    • (2003) N Engl J Med , vol.348 , pp. 255-256
    • Hacein-Bey-Abina, S.1    von Kalle, C.2    Schmidt, M.3
  • 56
    • 0142089747 scopus 로고    scopus 로고
    • Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
    • Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80(1-2):148-58.
    • (2003) Mol Genet Metab , vol.80 , Issue.1-2 , pp. 148-158
    • Raper, S.E.1    Chirmule, N.2    Lee, F.S.3
  • 57
    • 0024328536 scopus 로고
    • Altering the genome by homologous recombination
    • Capecchi MR. Altering the genome by homologous recombination. Science 1989;244:1288-92.
    • (1989) Science , vol.244 , pp. 1288-1292
    • Capecchi, M.R.1
  • 58
    • 79957829835 scopus 로고    scopus 로고
    • Gene therapy for primary immunodeficiencies: Looking ahead, toward gene correction
    • Pessach IM, Notarangelo LD. Gene therapy for primary immunodeficiencies: looking ahead, toward gene correction. J Allergy Clin Immunol 201;127:1344-50.
    • J Allergy Clin Immunol , vol.127 , pp. 1344-1350
    • Pessach, I.M.1    Notarangelo, L.D.2
  • 59
    • 27744530147 scopus 로고    scopus 로고
    • Zinc finger nucleases: Custom-designed molecular scissors for genome engineering of plant and mammalian cells
    • Durai S, Mani M, Kandavelou K, et al. Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells. Nucleic Acids Res 2005;33:5978-90.
    • (2005) Nucleic Acids Res , vol.33 , pp. 5978-5990
    • Durai, S.1    Mani, M.2    Kandavelou, K.3
  • 60
    • 37549030199 scopus 로고    scopus 로고
    • Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin
    • Hanna J, Wernig M, Markoulaki S, et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007;318(5858):1920-3.
    • (2007) Science , vol.318 , Issue.5858 , pp. 1920-1923
    • Hanna, J.1    Wernig, M.2    Markoulaki, S.3
  • 61
    • 0035861452 scopus 로고    scopus 로고
    • Correction in sickle cell disease in transgenic mouse models by gene therapy
    • Pawliuk R, Westerman KA, Fabry ME, et al. Correction in sickle cell disease in transgenic mouse models by gene therapy. Science 2001;294:2368-71.
    • (2001) Science , vol.294 , pp. 2368-2371
    • Pawliuk, R.1    Westerman, K.A.2    Fabry, M.E.3
  • 62
    • 80054987997 scopus 로고    scopus 로고
    • Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells
    • Yusa K, Rashid ST, Strick-Marchand H, et al. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature 2011;478:391-4.
    • (2011) Nature , vol.478 , pp. 391-394
    • Yusa, K.1    Rashid, S.T.2    Strick-Marchand, H.3
  • 63
    • 0034192227 scopus 로고    scopus 로고
    • Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin
    • Fakharzadeh SS, Zhang Y, Sarkar R, et al. Correction of the coagulation defect in hemophilia A mice through factor VIII expression in skin. Blood 2000;95:2799-805.
    • (2000) Blood , vol.95 , pp. 2799-2805
    • Fakharzadeh, S.S.1    Zhang, Y.2    Sarkar, R.3
  • 64
    • 0032005213 scopus 로고    scopus 로고
    • Human factor IX corrects the bleeding diathesis of mice with hemophilia B
    • Kung SH, Hagstrom JN, Cass D, et al. Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood 1998;91:784-90.
    • (1998) Blood , vol.91 , pp. 784-790
    • Kung, S.H.1    Hagstrom, J.N.2    Cass, D.3
  • 65
    • 58849113643 scopus 로고    scopus 로고
    • Phenotypic correction of murine hemophilia A using an iPS cell-based therapy
    • Xu D, Alipio Z, Fink LM, et al. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc Natl Acad Sci USA 2009;106(3):808-13.
    • (2009) Proc Natl Acad Sci USA , vol.106 , Issue.3 , pp. 808-813
    • Xu, D.1    Alipio, Z.2    Fink, L.M.3
  • 66
    • 77952600845 scopus 로고    scopus 로고
    • Susceptibility pathways in Fanconi's anemia and breast cancer
    • D'Andrea AD. Susceptibility pathways in Fanconi's anemia and breast cancer. N Engl J Med 2010;362:1909-19.
    • (2010) N Engl J Med , vol.362 , pp. 1909-1919
    • D'Andrea, A.D.1
  • 67
    • 0035379611 scopus 로고    scopus 로고
    • The emerging genetic and molecular basis of Fanconi anaemia
    • Joenje H, Patel KJ. The emerging genetic and molecular basis of Fanconi anaemia. Nat Rev Genet 2001;2:446-57.
    • (2001) Nat Rev Genet , vol.2 , pp. 446-457
    • Joenje, H.1    Patel, K.J.2
  • 68
    • 17744394476 scopus 로고    scopus 로고
    • Positional cloning of a novel Fanconi anemia gene, FANCD2
    • Timmers C, Taniguchi T, Hejna J, et al. Positional cloning of a novel Fanconi anemia gene, FANCD2. Mol Cell 2001;7:241-8.
    • (2001) Mol Cell , vol.7 , pp. 241-248
    • Timmers, C.1    Taniguchi, T.2    Hejna, J.3
  • 69
    • 18444362122 scopus 로고    scopus 로고
    • Biallelic inactivation of BRCA2 in Fanconi anemia
    • Howlett NG, Taniguchi T, Olson S, et al. Biallelic inactivation of BRCA2 in Fanconi anemia. Science 2002;297:606-9.
    • (2002) Science , vol.297 , pp. 606-609
    • Howlett, N.G.1    Taniguchi, T.2    Olson, S.3
  • 70
    • 0037108187 scopus 로고    scopus 로고
    • Gene therapy of Fanconi anemia: Preclinical efficacy using lentiviral vectors
    • Galimi F, Noll M, Kanazawa Y, et al. Gene therapy of Fanconi anemia: preclinical efficacy using lentiviral vectors. Blood 2002;100:2732-6.
    • (2002) Blood , vol.100 , pp. 2732-2736
    • Galimi, F.1    Noll, M.2    Kanazawa, Y.3
  • 71
    • 77949427674 scopus 로고    scopus 로고
    • A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells
    • Raya A, Rodriguez-Piza I, Navarro S, et al. A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells. Nat Protoc 2010;5(4): 647-60.
    • (2010) Nat Protoc , vol.5 , Issue.4 , pp. 647-660
    • Raya, A.1    Rodriguez-Piza, I.2    Navarro, S.3
  • 72
    • 55049122748 scopus 로고    scopus 로고
    • Reprogramming of neural progenitor cells into induced pluripotent stem cells in the absence of exogenous Sox2 expression
    • Eminli S, Utikal J, Arnold K, et al. Reprogramming of neural progenitor cells into induced pluripotent stem cells in the absence of exogenous Sox2 expression. Stem Cells 2008;26:2467-74.
    • (2008) Stem Cells , vol.26 , pp. 2467-2474
    • Eminli, S.1    Utikal, J.2    Arnold, K.3
  • 73
    • 37349040710 scopus 로고    scopus 로고
    • Induction of pluripotent stem cells from mouse fibroblasts by four transcription factors
    • Yamanaka S. Induction of pluripotent stem cells from mouse fibroblasts by four transcription factors. Cell Prolif 2008;41(1):51-6.
    • (2008) Cell Prolif , vol.41 , Issue.1 , pp. 51-56
    • Yamanaka, S.1
  • 74
    • 79952258224 scopus 로고    scopus 로고
    • Somatic coding mutations in human induced pluripotent stem cells
    • Gore A, Li Z, Fung HL, et al. Somatic coding mutations in human induced pluripotent stem cells. Nature 2011;471(7336):63-7.
    • (2011) Nature , vol.471 , Issue.7336 , pp. 63-67
    • Gore, A.1    Li, Z.2    Fung, H.L.3
  • 75
    • 78650971216 scopus 로고    scopus 로고
    • Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture
    • Laurent LC, Ulitsky I, Slavin I, et al. Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. Cell Stem Cell 2011;8:106-18.
    • (2011) Cell Stem Cell , vol.8 , pp. 106-118
    • Laurent, L.C.1    Ulitsky, I.2    Slavin, I.3
  • 76
    • 79952264847 scopus 로고    scopus 로고
    • Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells
    • Lister R, Pelizzola M, Kida YS, et al. Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells. Nature 2011;471(7336):68-73.
    • (2011) Nature , vol.471 , Issue.7336 , pp. 68-73
    • Lister, R.1    Pelizzola, M.2    Kida, Y.S.3
  • 77
    • 63649132974 scopus 로고    scopus 로고
    • Correction of murine sickle cell disease using γ-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin
    • Pestina TI, Hargrove PW, Jay D, et al. Correction of murine sickle cell disease using γ-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin. Mol Ther 2009;17(2):245-52.
    • (2009) Mol Ther , vol.17 , Issue.2 , pp. 245-252
    • Pestina, T.I.1    Hargrove, P.W.2    Jay, D.3
  • 78
    • 70450247528 scopus 로고    scopus 로고
    • Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders
    • Ye ZH, Zhan HC, Mali P, et al. Human-induced pluripotent stem cells from blood cells of healthy donors and patients with acquired blood disorders. Blood 2009;114:5473-80.
    • (2009) Blood , vol.114 , pp. 5473-5480
    • Ye, Z.H.1    Zhan, H.C.2    Mali, P.3
  • 79
    • 77955814966 scopus 로고    scopus 로고
    • Reversal of hyperglycemia in diabetic mouse models using induced-pluripotent stem (iPS)-derived pancreatic β-like cells
    • Alipio Z, Liao WB, Roemer EJ, et al. Reversal of hyperglycemia in diabetic mouse models using induced-pluripotent stem (iPS)-derived pancreatic β-like cells. Proc Natl Acad Sci USA 2010;107(30):13426-134-31.
    • (2010) Proc Natl Acad Sci USA , vol.107 , Issue.30 , pp. 13426-13431
    • Alipio, Z.1    Liao, W.B.2    Roemer, E.J.3
  • 80
    • 77956354416 scopus 로고    scopus 로고
    • Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells
    • Rashid ST, Corbineau S, Hannan N, et al. Modeling inherited metabolic disorders of the liver using human induced pluripotent stem cells. J Clin Invest 2010;120:3127-36.
    • (2010) J Clin Invest , vol.120 , pp. 3127-3136
    • Rashid, S.T.1    Corbineau, S.2    Hannan, N.3
  • 81
    • 77957347780 scopus 로고    scopus 로고
    • Generation of liver disease-specific induced pluripotent stem cells along with efficient differentiation to functional hepatocyte-like cells
    • Ghodsizadeh A, Taei A, Totonchi M, et al. Generation of liver disease-specific induced pluripotent stem cells along with efficient differentiation to functional hepatocyte-like cells. Stem Cell Rev 2010; 6:622-32.
    • (2010) Stem Cell Rev , vol.6 , pp. 622-632
    • Ghodsizadeh, A.1    Taei, A.2    Totonchi, M.3
  • 82
    • 80052531213 scopus 로고    scopus 로고
    • Generation of induced pluripotent stem (iPS) cells derived from a murine model of Pompe disease and differentiation of Pompe-iPS cells into skeletal muscle cells
    • Kawagoe S, Higuchi T, Meng XL, et al. Generation of induced pluripotent stem (iPS) cells derived from a murine model of Pompe disease and differentiation of Pompe-iPS cells into skeletal muscle cells. Mol Genet Metab 2011;104:123-8.
    • (2011) Mol Genet Metab , vol.104 , pp. 123-128
    • Kawagoe, S.1    Higuchi, T.2    Meng, X.L.3
  • 83
    • 79960831061 scopus 로고    scopus 로고
    • Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin
    • Zhang SQ, Chen S, Li W, et al. Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin. Hum Mol Genet 2011;20(16):3176-87.
    • (2011) Hum Mol Genet , vol.20 , Issue.16 , pp. 3176-3187
    • Zhang, S.Q.1    Chen, S.2    Li, W.3
  • 84
    • 78751684511 scopus 로고    scopus 로고
    • Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)
    • Tolar J, Park IH, Xia L, et al. Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome). Blood 2011;117:839-47.
    • (2011) Blood , vol.117 , pp. 839-847
    • Tolar, J.1    Park, I.H.2    Xia, L.3
  • 85
    • 58249110796 scopus 로고    scopus 로고
    • Induced pluripotent stem cells from a spinal muscular atrophy patient
    • Ebert AD, Yu J, Rose FF Jr, et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009;457(7227):277-80.
    • (2009) Nature , vol.457 , Issue.7227 , pp. 277-280
    • Ebert, A.D.1    Yu, J.2    Rose Jr., F.F.3
  • 86
    • 78149488365 scopus 로고    scopus 로고
    • A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells
    • Marchetto MCN, Carromeu C, Acab A, et al. A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells. Cell 2010;143:527-39.
    • (2010) Cell , vol.143 , pp. 527-539
    • Marchetto, M.C.N.1    Carromeu, C.2    Acab, A.3
  • 87
    • 79955884485 scopus 로고    scopus 로고
    • Modelling schizophrenia using human induced pluripotent stem cells
    • Brennand KJ, Simone A, Jou J, et al. Modelling schizophrenia using human induced pluripotent stem cells. Nature 2011;473(7346):221-7.
    • (2011) Nature , vol.473 , Issue.7346 , pp. 221-227
    • Brennand, K.J.1    Simone, A.2    Jou, J.3
  • 88
    • 61349100729 scopus 로고    scopus 로고
    • Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors
    • Soldner F, Hockemeyer D, Beard C, et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 2009;136:964-77.
    • (2009) Cell , vol.136 , pp. 964-977
    • Soldner, F.1    Hockemeyer, D.2    Beard, C.3
  • 89
    • 81155131691 scopus 로고    scopus 로고
    • SNCA triplication Parkinson's patient's iPSC-derived DA neurons accumulate α-synuclein and are susceptible to oxidative stress
    • Byers B, Cord B, Nguyen HN, et al. SNCA triplication Parkinson's patient's iPSC-derived DA neurons accumulate α-synuclein and are susceptible to oxidative stress. PLoS One 2011;6(11):e26159.
    • (2011) PLoS One , vol.6 , Issue.11
    • Byers, B.1    Cord, B.2    Nguyen, H.N.3
  • 90
    • 80053628732 scopus 로고    scopus 로고
    • Grafted human-induced pluripotent stem-cell-derived neurospheres promote motor functional recovery after spinal cord injury in mice
    • Nori S, Okada Y, Yasuda A, et al. Grafted human-induced pluripotent stem-cell-derived neurospheres promote motor functional recovery after spinal cord injury in mice. Proc Natl Acad Sci USA 2011;108(40):16825-30.
    • (2011) Proc Natl Acad Sci USA , vol.108 , Issue.40 , pp. 16825-16830
    • Nori, S.1    Okada, Y.2    Yasuda, A.3
  • 91
    • 84856956771 scopus 로고    scopus 로고
    • Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells
    • Israel MA, Yuan SH, Bardy C, et al. Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells. Nature 2012;482(7384):216-20.
    • (2012) Nature , vol.482 , Issue.7384 , pp. 216-220
    • Israel, M.A.1    Yuan, S.H.2    Bardy, C.3
  • 92
    • 77953443251 scopus 로고    scopus 로고
    • Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome
    • Carvajal-Vergara X, Sevilla A, D'Souza SL, et al. Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome. Nature 2010;465(7299):808-12.
    • (2010) Nature , vol.465 , Issue.7299 , pp. 808-812
    • Carvajal-Vergara, X.1    Sevilla, A.2    D'Souza, S.L.3
  • 93
    • 79952438377 scopus 로고    scopus 로고
    • Using induced pluripotent stem cells to investigate cardiac phenotypes in Timothy syndrome
    • Yazawa M, Hsueh B, Jia X, et al. Using induced pluripotent stem cells to investigate cardiac phenotypes in Timothy syndrome. Nature 2011;471(7337):230-4.
    • (2011) Nature , vol.471 , Issue.7337 , pp. 230-234
    • Yazawa, M.1    Hsueh, B.2    Jia, X.3
  • 94
    • 79959282291 scopus 로고    scopus 로고
    • Telomere shortening and loss of self-renewal in dyskeratosis congenita induced pluripotent stem cells
    • Batista LF, Pech MF, Zhong MF, et al. Telomere shortening and loss of self-renewal in dyskeratosis congenita induced pluripotent stem cells. Nature 2011;474(7351):399-402.
    • (2011) Nature , vol.474 , Issue.7351 , pp. 399-402
    • Batista, L.F.1    Pech, M.F.2    Zhong, M.F.3
  • 95
    • 79951849153 scopus 로고    scopus 로고
    • Modeling retinal degeneration using patient-specific induced pluripotent stem cells
    • Jin ZB, Okamoto S, Osakada F, et al. Modeling retinal degeneration using patient-specific induced pluripotent stem cells. PLoS One 2011;6:e17084.
    • (2011) PLoS One , vol.6
    • Jin, Z.B.1    Okamoto, S.2    Osakada, F.3
  • 96
    • 79957696056 scopus 로고    scopus 로고
    • Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells
    • Itoh M, Kiuru M, Cairo MS, et al. Generation of keratinocytes from normal and recessive dystrophic epidermolysis bullosa-induced pluripotent stem cells. Proc Natl Acad Sci USA 2011;108(21):8797-802.
    • (2011) Proc Natl Acad Sci USA , vol.108 , Issue.21 , pp. 8797-8802
    • Itoh, M.1    Kiuru, M.2    Cairo, M.S.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.