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Volumn 128, Issue 1, 2011, Pages 8-13

Induced human pluripotent stem cells and advanced therapies: Future perspectives for the treatment of haemophilia?

Author keywords

Advanced therapies; Cell therapy; Haemophilia; In vitro reprogramming; Induced pluripotent stem cells; iPSCs

Indexed keywords

ADENOVIRUS VECTOR; KRUPPEL LIKE FACTOR 4; LENTIVIRUS VECTOR; MYC PROTEIN; OCTAMER TRANSCRIPTION FACTOR 4; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9; RETROVIRUS VECTOR; TRANSCRIPTION FACTOR SOX2;

EID: 79958035838     PISSN: 00493848     EISSN: 18792472     Source Type: Journal    
DOI: 10.1016/j.thromres.2011.01.010     Document Type: Review
Times cited : (12)

References (60)
  • 1
    • 34249863603 scopus 로고    scopus 로고
    • Strategies and New Developments in the Generation of Patient-Specific Pluripotent Stem Cells
    • DOI 10.1016/j.stem.2007.05.012, PII S1934590907000185
    • Yamanaka S. Strategies and new developments in the generation of patient-specific pluripotent stem cells. Cell Stem Cell 2007;1:39-49. (Pubitemid 46865811)
    • (2007) Cell Stem Cell , vol.1 , Issue.1 , pp. 39-49
    • Yamanaka, S.1
  • 2
    • 33747195353 scopus 로고    scopus 로고
    • Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors
    • DOI 10.1016/j.cell.2006.07.024, PII S0092867406009767
    • Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006;126:663-76. (Pubitemid 44233629)
    • (2006) Cell , vol.126 , Issue.4 , pp. 663-676
    • Takahashi, K.1    Yamanaka, S.2
  • 3
    • 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
  • 4
    • 65649116572 scopus 로고    scopus 로고
    • Human induced pluripotent stem cells free of vector and transgene sequences
    • Yu J, Hu K, Smuga-Otto K, et al. Human induced pluripotent stem cells free of vector and transgene sequences. Science 2009;324:797-801.
    • (2009) Science , vol.324 , pp. 797-801
    • Yu, J.1    Hu, K.2    Smuga-Otto, K.3
  • 5
    • 38049187707 scopus 로고    scopus 로고
    • Reprogramming of human somatic cells to pluripotency with defined factors
    • Park IH, Zhao R, West JA, et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008;451:141-6.
    • (2008) Nature , vol.451 , pp. 141-146
    • Park, I.H.1    Zhao, R.2    West, J.A.3
  • 6
    • 77749320143 scopus 로고    scopus 로고
    • Generation of mouse-induced pluripotent stem cells with plasmid vectors
    • Okita K, Hong H, Takahashi K, et al. Generation of mouse-induced pluripotent stem cells with plasmid vectors. Nat Protoc 2010;5:418-28.
    • (2010) Nat Protoc , vol.5 , pp. 418-428
    • Okita, K.1    Hong, H.2    Takahashi, K.3
  • 7
    • 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
  • 8
    • 64749083939 scopus 로고    scopus 로고
    • PiggyBac transposition reprograms fibroblasts to induced pluripotent stem cells
    • Woltjen K, Michael IP, Mohseni P, et al. PiggyBac 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
  • 9
    • 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
  • 10
    • 70350732790 scopus 로고    scopus 로고
    • A chemical platform for improved induction of human iPSCs
    • Lin T, Ambasudhan R, Yuan X, et al. A chemical platform for improved induction of human iPSCs. Nat Methods 2009;6:805-8.
    • (2009) Nat Methods , vol.6 , pp. 805-808
    • Lin, T.1    Ambasudhan, R.2    Yuan, X.3
  • 12
    • 25644456543 scopus 로고    scopus 로고
    • Available atWhite paper: Washington, D.C; last accessed on 23 January 2011
    • Alternative sources of human pluripotent stem cells. . Available atWhite paper: The President's Council on Bioethics. Washington, D.C; 2005 http://bioethics.georgetown.edu/pcbe/reports/white-paper/alternative-sources- white-paper.pdf. (last accessed on 23 January 2011).
    • (2005) Alternative Sources of Human Pluripotent Stem Cells
  • 13
    • 67650229354 scopus 로고    scopus 로고
    • Available atNational Research Council and Institute of Medicine. National Academy Press; last accessed on 23 January 2011
    • 2008 Amendments to the National Academies' guidelines for human embryonic stem cell research. . Available atNational Research Council and Institute of Medicine. National Academy Press; 2008 http://www.nap.edu/openbook.php?record- id=12260&page=1. (last accessed on 23 January 2011).
    • (2008) 2008 Amendments to the National Academies' Guidelines for Human Embryonic Stem Cell Research
  • 14
    • 77957334627 scopus 로고    scopus 로고
    • Identification and classification of chromosomal aberrations in human induced pluripotent stem cells
    • Mayshar Y, Ben-David U, Lavon N, et al. Identification and classification of chromosomal aberrations in human induced pluripotent stem cells. Cell Stem Cell 2010;7:521-31.
    • (2010) Cell Stem Cell , vol.7 , pp. 521-531
    • Mayshar, Y.1    Ben-David, U.2    Lavon, N.3
  • 15
    • 70349315647 scopus 로고    scopus 로고
    • Induced human pluripotent stem cells: Promises and open questions
    • Rolletschek A, Wobus AM. Induced human pluripotent stem cells: promises and open questions. Biol Chem 2009;390:845-9.
    • (2009) Biol Chem , vol.390 , pp. 845-849
    • Rolletschek, A.1    Wobus, A.M.2
  • 16
    • 60649084367 scopus 로고    scopus 로고
    • Induced pluripotent stem cells: Current progress and potential for regenerative medicine
    • Amabile G, Meissner A. Induced pluripotent stem cells: current progress and potential for regenerative medicine. Trends Mol Med 2009;15:59-68.
    • (2009) Trends Mol Med , vol.15 , pp. 59-68
    • Amabile, G.1    Meissner, A.2
  • 17
    • 64249157351 scopus 로고    scopus 로고
    • Epigenetic reprogramming and induced pluripotency
    • Hochedlinger K, Plath K. Epigenetic reprogramming and induced pluripotency. Development 2009;136:509-23.
    • (2009) Development , vol.136 , pp. 509-523
    • Hochedlinger, K.1    Plath, K.2
  • 18
    • 57649128308 scopus 로고    scopus 로고
    • Generation of insulin-secreting islet-like clusters from human skin fibroblasts
    • Tateishi K, He J, Taranova O, et al. Generation of insulin-secreting islet-like clusters from human skin fibroblasts. J Biol Chem 2008;283:31601-7.
    • (2008) J Biol Chem , vol.283 , pp. 31601-31607
    • Tateishi, K.1    He, J.2    Taranova, O.3
  • 19
    • 50149098605 scopus 로고    scopus 로고
    • Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons
    • Dimos JT, Rodolfa KT, Niakan KK, et al. Induced pluripotent stem cells generated from patients with ALS 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
  • 20
    • 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;134:877-86.
    • (2008) Cell , vol.134 , pp. 877-886
    • Park, I.H.1    Arora, N.2    Huo, H.3
  • 21
    • 58249110796 scopus 로고    scopus 로고
    • Induced pluripotent stem cells from a spinal muscular atrophy patient
    • Ebert AD, Yu J, Rose FF, et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009;457:277-80.
    • (2009) Nature , vol.457 , pp. 277-280
    • Ebert, A.D.1    Yu, J.2    Rose, F.F.3
  • 22
    • 60149107033 scopus 로고    scopus 로고
    • In vitro electrophysiological drug testing using human embryonic stem cell derived cardiomyocytes
    • Caspi O, Itzhaki I, Arbel G, et al. In vitro electrophysiological drug testing using human embryonic stem cell derived cardiomyocytes. Stem Cells Dev 2009;18:161-72.
    • (2009) Stem Cells Dev , vol.18 , pp. 161-172
    • Caspi, O.1    Itzhaki, I.2    Arbel, G.3
  • 24
    • 37549030199 scopus 로고    scopus 로고
    • Treatment of sickle cell anemia mouse model with iPSCs cells generated from autologous skin
    • Hanna J, Wernig M, Markoulaki S, et al. Treatment of sickle cell anemia mouse model with iPSCs cells generated from autologous skin. Science 2007;318:1920-3.
    • (2007) Science , vol.318 , pp. 1920-1923
    • Hanna, J.1    Wernig, M.2    Markoulaki, S.3
  • 25
    • 49649096192 scopus 로고    scopus 로고
    • Generation of functional murine cardiac myocytes from induced pluripotent stem cells
    • Mauritz C, Schwanke K, Reppel M, et al. Generation of functional murine cardiac myocytes from induced pluripotent stem cells. Circulation 2008;118:507-17.
    • (2008) Circulation , vol.118 , pp. 507-517
    • Mauritz, C.1    Schwanke, K.2    Reppel, M.3
  • 26
    • 61949300076 scopus 로고    scopus 로고
    • Functional cardiomyocytes derived from human induced pluripotent stem cells
    • Zhang J, Wilson GF, Soerens AG, et al. Functional cardiomyocytes derived from human induced pluripotent stem cells. Circ Res 2009;104:e30-41.
    • (2009) Circ Res , vol.104
    • Zhang, J.1    Wilson, G.F.2    Soerens, A.G.3
  • 27
    • 68049137505 scopus 로고    scopus 로고
    • Induced pluripotent stem cells: An emerging technology platform and the Gartner hype cycle
    • Mason C, Manzotti E. Induced pluripotent stem cells: an emerging technology platform and the Gartner hype cycle. Regen Med 2009;4:329-31.
    • (2009) Regen Med , vol.4 , pp. 329-331
    • Mason, C.1    Manzotti, E.2
  • 28
    • 68049137797 scopus 로고    scopus 로고
    • Induced pluripotent stem cells: Reprogrammed without a trace
    • Nelson TJ, Terzic A. Induced pluripotent stem cells: reprogrammed without a trace. Regen Med 2009;4:333-55.
    • (2009) Regen Med , vol.4 , pp. 333-355
    • Nelson, T.J.1    Terzic, A.2
  • 29
    • 66149110999 scopus 로고    scopus 로고
    • Protein replacement therapy and gene transfer in canine models of haemophilia A, haemophilia B, von willebrand disease, and factor VII deficiency
    • Nichols TC, Dillow AM, Franck HW, et al. Protein replacement therapy and gene transfer in canine models of haemophilia A, haemophilia B, von willebrand disease, and factor VII deficiency. ILAR J 2009;50:144-67.
    • (2009) ILAR J , vol.50 , pp. 144-167
    • Nichols, T.C.1    Dillow, A.M.2    Franck, H.W.3
  • 30
    • 44849127181 scopus 로고    scopus 로고
    • Development of gene therapy for blood disorders
    • Nienhuis AW. Development of gene therapy for blood disorders. Blood 2009;111:4431-44.
    • (2009) Blood , vol.111 , pp. 4431-4444
    • Nienhuis, A.W.1
  • 31
    • 0034804403 scopus 로고    scopus 로고
    • Gene therapy for haemophilia: The end of a "royal pathology" in the third millennium?
    • Liras A. Gene therapy for haemophilia: The end of a "royal pathology" in the third millennium? Haemophilia 2001;7:441-5.
    • (2001) Haemophilia , vol.7 , pp. 441-445
    • Liras, A.1
  • 32
    • 65449140491 scopus 로고    scopus 로고
    • Gene therapy for haemophilia...yes, but...with non-viral vectors?
    • Liras A, Olmedillas S. Gene therapy for haemophilia...yes, but...with non-viral vectors? Haemophilia 2009;15:811-6.
    • (2009) Haemophilia , vol.15 , pp. 811-816
    • Liras, A.1    Olmedillas, S.2
  • 33
    • 63149161694 scopus 로고    scopus 로고
    • Gene therapy for hemophilia: Clinical trials and technical tribulations
    • Viiala NO, Larsen SR, Rasko JE. Gene therapy for hemophilia: clinical trials and technical tribulations. Semin Thromb Hemost 2009;35:81-92.
    • (2009) Semin Thromb Hemost , vol.35 , pp. 81-92
    • Viiala, N.O.1    Larsen, S.R.2    Rasko, J.E.3
  • 35
    • 75749084514 scopus 로고    scopus 로고
    • Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector
    • Jeon HJ, Oh TK, Kim OH, et al. Delivery of factor VIII gene into skeletal muscle cells using lentiviral vector. Yonsei Med J 2010;51:52-7.
    • (2010) Yonsei Med J , vol.51 , pp. 52-57
    • Jeon, H.J.1    Oh, T.K.2    Kim, O.H.3
  • 36
    • 21244476011 scopus 로고    scopus 로고
    • AAV-mediated gene transfer for treatment of hemophilia
    • DOI 10.2174/1566523054065048
    • Wang L, Herzog RW. AAV-mediated gene transfer for treatment of hemophilia. Curr Gene Ther 2005;5:349-60. (Pubitemid 40895019)
    • (2005) Current Gene Therapy , vol.5 , Issue.3 , pp. 349-360
    • Wang, L.1    Herzog, R.W.2
  • 37
    • 77954245040 scopus 로고    scopus 로고
    • Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B
    • Arruda VR, Stedman HH, Haurigot V, et al. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood 2010;115:4678-88.
    • (2010) Blood , vol.115 , pp. 4678-4688
    • Arruda, V.R.1    Stedman, H.H.2    Haurigot, V.3
  • 39
    • 77949674544 scopus 로고    scopus 로고
    • Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: Therapeutic expression of factor IX at reduced vector doses
    • Jayandharan GR, Zhong L, Sack BK, et al. Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses. Hum Gene Ther 2010;21:271-83.
    • (2010) Hum Gene Ther , vol.21 , pp. 271-283
    • Jayandharan, G.R.1    Zhong, L.2    Sack, B.K.3
  • 40
    • 77954244683 scopus 로고    scopus 로고
    • Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs
    • Haurigot V, Mingozzi F, Buchlis G, et al. Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs. Mol Ther 2010;18:1318-29.
    • (2010) Mol Ther , vol.18 , pp. 1318-1329
    • Haurigot, V.1    Mingozzi, F.2    Buchlis, G.3
  • 41
    • 78650653228 scopus 로고    scopus 로고
    • In vivo efficacy of platelet-delivered, high specific activity factor VIII variants
    • Greene TK, Wang C, Hirsch JD, et al. In vivo efficacy of platelet-delivered, high specific activity factor VIII variants. Blood 2010;116:6114-22.
    • (2010) Blood , vol.116 , pp. 6114-6122
    • Greene, T.K.1    Wang, C.2    Hirsch, J.D.3
  • 43
    • 77955921912 scopus 로고    scopus 로고
    • Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice
    • Zhang G, Shi Q, Fahs SA, et al. Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice. Blood 2010;116:1235-43.
    • (2010) Blood , vol.116 , pp. 1235-1243
    • Zhang, G.1    Shi, Q.2    Fahs, S.A.3
  • 47
    • 77954833516 scopus 로고    scopus 로고
    • Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
    • Hacein-Bey-Abina S, Hauer J, Lim A, et al. Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency. N Engl J Med 2010;363:355-64.
    • (2010) N Engl J Med , vol.363 , pp. 355-364
    • Hacein-Bey-Abina, S.1    Hauer, J.2    Lim, A.3
  • 48
    • 59449098985 scopus 로고    scopus 로고
    • Gene therapy for immunodeficiency due to adenosine deaminase deficiency
    • Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009;360:447-58.
    • (2009) N Engl J Med , vol.360 , pp. 447-458
    • Aiuti, A.1    Cattaneo, F.2    Galimberti, S.3
  • 49
    • 77950874920 scopus 로고    scopus 로고
    • Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A
    • Ide LM, Iwakoshi NN, Gangadharan B, et al. Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A. J Gene Med 2010;12:333-44.
    • (2010) J Gene Med , vol.12 , pp. 333-344
    • Ide, L.M.1    Iwakoshi, N.N.2    Gangadharan, B.3
  • 50
    • 77954244026 scopus 로고    scopus 로고
    • Biosafety assessment of site-directed transgene integration in human umbilical cord-lining cells
    • Sivalingam J, Krishnan S, Ng WH, et al. Biosafety assessment of site-directed transgene integration in human umbilical cord-lining cells. Mol Ther 2010;18:1346-56.
    • (2010) Mol Ther , vol.18 , pp. 1346-1356
    • Sivalingam, J.1    Krishnan, S.2    Ng, W.H.3
  • 52
    • 40549129676 scopus 로고    scopus 로고
    • Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice
    • DOI 10.1172/JCI32748
    • Follenzi A, Benten D, Novikoff P, et al. Transplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A mice. J Clin Invest 2008;118:935-45. (Pubitemid 351364601)
    • (2008) Journal of Clinical Investigation , vol.118 , Issue.3 , pp. 935-945
    • Follenzi, A.1    Benten, D.2    Novikoff, P.3    Faulkner, L.4    Raut, S.5    Gupta, S.6
  • 53
    • 58849113643 scopus 로고    scopus 로고
    • Phenotypic correction of murine hemophilia A using an iPSCs cell-based therapy
    • Xu D, Alipio Z, Fink LM, et al. Phenotypic correction of murine hemophilia A using an iPSCs cell-based therapy. Proc Natl Acad Sci USA 2009;106:808-13.
    • (2009) Proc Natl Acad Sci USA , vol.106 , pp. 808-813
    • Xu, D.1    Alipio, Z.2    Fink, L.M.3
  • 54
    • 73349121646 scopus 로고    scopus 로고
    • The therapeutic effect of bone marrow-derived liver cells in the phenotypic correction of murine hemophilia A
    • Yadav N, Kanjirakkuzhiyil S, Kumar S, et al. The therapeutic effect of bone marrow-derived liver cells in the phenotypic correction of murine hemophilia A. Blood 2009;114:4552-61.
    • (2009) Blood , vol.114 , pp. 4552-4561
    • Yadav, N.1    Kanjirakkuzhiyil, S.2    Kumar, S.3
  • 56
    • 77955701842 scopus 로고    scopus 로고
    • Evolution of induced pluripotent stem cell technology
    • Zhou H, Ding S. Evolution of induced pluripotent stem cell technology. Curr Opin Hematol 2010;17:276-80.
    • (2010) Curr Opin Hematol , vol.17 , pp. 276-280
    • Zhou, H.1    Ding, S.2
  • 57
    • 77950675023 scopus 로고    scopus 로고
    • iPS cell technology in regenerative medicine
    • Lengner CJ. iPS cell technology in regenerative medicine. Ann NY Acad Sci 2010;1192:38-44.
    • (2010) Ann NY Acad Sci , vol.1192 , pp. 38-44
    • Lengner, C.J.1
  • 58
    • 77949328902 scopus 로고    scopus 로고
    • Pluripotent human stem cells: A novel tool in drug discovery
    • Phillips BW, Crook JM. Pluripotent human stem cells: A novel tool in drug discovery. BioDrugs 2010;24:99-108.
    • (2010) BioDrugs , vol.24 , pp. 99-108
    • Phillips, B.W.1    Crook, J.M.2
  • 59
    • 77957773731 scopus 로고    scopus 로고
    • Red blood cells generation from human induced pluripotent stem cells: Perspectives for transfusion medicine
    • Lapillonne H, Kobari L, Mazurier C, et al. Red blood cells generation from human induced pluripotent stem cells: perspectives for transfusion medicine. Haematologica 2010;95:1651-9.
    • (2010) Haematologica , vol.95 , pp. 1651-1659
    • Lapillonne, H.1    Kobari, L.2    Mazurier, C.3
  • 60
    • 78649810923 scopus 로고    scopus 로고
    • Gene therapy, gene targeting and induced pluripotent stemcells: Applications in monogenic disease treatment
    • Wong GK, Chiu AT. Gene therapy, gene targeting and induced pluripotent stemcells: Applications in monogenic disease treatment. Biotechnol Adv 2011;29:1-10.
    • (2011) Biotechnol Adv , vol.29 , pp. 1-10
    • Wong, G.K.1    Chiu, A.T.2


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