CoChat
Sign Up
Cited by 54

The DOI matches its Crossref record; 18 of the 18 references it cites were found in a registry or an index.

Hematopoietic stem cell engineering at a crossroads

Rivière, Isabelle, Dunbar, Cynthia E., Sadelain, Michel·2012·Blood
DOI 10.1182/blood-2011-09-349993
Open this paper in CoChat — free account

Abstract

The genetic engineering of hematopoietic stem cells is the basis for potentially treating a large array of hereditary and acquired diseases, and stands as the paradigm for stem cell engineering in general. Recent clinical reports support the formidable promise of this approach but also highlight the limitations of the technologies used to date, which have on occasion resulted in clonal expansion, myelodysplasia, or leukemogenesis. New research directions, predicated on improved vector designs, targeted gene delivery or the therapeutic use of pluripotent stem cells, herald the advent of safer and more effective hematopoietic stem cell therapies that may transform medical practice. In this review, we place these recent advances in perspective, emphasizing the solutions emerging from a wave of new technologies and highlighting the challenges that lie ahead.

Overview

Integrity signals

Registry

Crossref · 10.1182/blood-2011-09-349993

Cites

18 of 18 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Verification

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Citations

Cites

Show all 18 references

Research works printed but not linked: 123

  1. Dunbar, 1996, Gene transfer to hematopoietic stem cells: implications for gene therapy of human disease., Annu Rev Med, 47, 11, 10.1146/annurev.med.47.1.11 · DOI
  2. Kohn, 2001, Gene therapy for genetic haematological disorders and immunodeficiencies., J Intern Med, 249, 379, 10.1046/j.1365-2796.2001.00803.x · DOI
  3. Nienhuis, 2006, Genotoxicity of retroviral integration in hematopoietic cells., Mol Ther, 13, 1031, 10.1016/j.ymthe.2006.03.001 · DOI · Paper page
  4. Kustikova, 2010, The genomic risk of somatic gene therapy., Semin Cancer Biol, 20, 269, 10.1016/j.semcancer.2010.06.003 · DOI
  5. Barese, 2011, Contributions of gene marking to cell and gene therapies., Hum Gene Ther, 22, 659, 10.1089/hum.2010.237 · DOI
  6. Miller, 1989, Improved retroviral vectors for gene transfer and expression., Biotechniques, 7, 980
  7. Riviere, 1995, Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells., Proc Natl Acad Sci U S A, 92, 6733, 10.1073/pnas.92.15.6733 · DOI
  8. Hildinger, 1998, FMEV vectors: both retroviral long terminal repeat and leader are important for high expression in transduced hematopoietic cells., Gene Ther, 5, 1575, 10.1038/sj.gt.3300759 · DOI
  9. Aiuti, 2007, Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy., J Clin Invest, 117, 2233, 10.1172/JCI31666 · DOI
  10. Schwarzwaelder, 2007, Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo., J Clin Invest, 117, 2241, 10.1172/JCI31661 · DOI
  11. Boztug, 2010, Stem-cell gene therapy for the Wiskott-Aldrich syndrome., N Engl J Med, 363, 1918, 10.1056/NEJMoa1003548 · DOI · Paper page
  12. Gaspar, 2011, Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction., Sci Transl Med, 3, 97ra80, 10.1126/scitranslmed.3002716 · DOI
  13. Gaspar, 2011, Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency., Sci Transl Med, 3, 97ra79, 10.1126/scitranslmed.3002715 · DOI
  14. Kohn, 2009, Gene therapy fulfilling its promise., N Engl J Med, 360, 518, 10.1056/NEJMe0809614 · DOI
  15. Ott, 2006, Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1., Nat Med, 12, 401, 10.1038/nm1393 · DOI · Paper page
  16. Rosenbaum, 2008, Granulocyte colony-stimulating factor-based stem cell mobilization in patients with sickle cell disease., Biol Blood Marrow Transplant, 14, 719, 10.1016/j.bbmt.2008.03.001 · DOI
  17. Gothot, 1998, Cell cycle-related changes in repopulating capacity of human mobilized peripheral blood CD34(+) cells in non-obese diabetic/severe combined immune-deficient mice., Blood, 92, 2641, 10.1182/blood.V92.8.2641 · DOI
  18. Takatoku, 2001, Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates., J Clin Invest, 108, 447, 10.1172/JCI12593 · DOI
  19. May, 2000, Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin., Nature, 406, 82, 10.1038/35017565 · DOI · Paper page
  20. Persons, 2010, The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in beta-thalassemia patients., Ann N Y Acad Sci, 1202, 69, 10.1111/j.1749-6632.2010.05581.x · DOI
  21. Hanawa, 2009, Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element., Mol Ther, 17, 667, 10.1038/mt.2009.1 · DOI
  22. Urbinati, 2009, Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3′LTR., Mol Ther, 17, 1527, 10.1038/mt.2009.89 · DOI
  23. Rivella, 1998, Genetic treatment of severe hemoglobinopathies: the combat against transgene variegation and transgene silencing., Semin Hematol, 35, 112
  24. Bestor, 2000, Gene silencing as a threat to the success of gene therapy., J Clin Invest, 105, 409, 10.1172/JCI9459 · DOI
  25. Ellis, 2005, Silencing and variegation of gammaretrovirus and lentivirus vectors., Hum Gene Ther, 16, 1241, 10.1089/hum.2005.16.1241 · DOI · Paper page
  26. Heim, 2000, Hematopoietic stem cell gene therapy: towards clinically significant gene transfer efficiency., Immunol Rev, 178, 29, 10.1034/j.1600-065X.2000.17802.x · DOI
  27. Stein, 2010, Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease., Nat Med, 16, 198, 10.1038/nm.2088 · DOI · Paper page
  28. Kim, 2009, Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells., Blood, 113, 5434, 10.1182/blood-2008-10-185199 · DOI
  29. Beard, 2006, Efficient transduction and engraftment of G-CSF-mobilized peripheral blood CD34+ cells in nonhuman primates using GALV-pseudotyped gammaretroviral vectors., Mol Ther, 14, 212, 10.1016/j.ymthe.2006.01.016 · DOI
  30. Brenner, 1993, Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients., Lancet, 342, 1134, 10.1016/0140-6736(93)92122-A · DOI
  31. Mitchell, 2004, Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences., PLoS Biol, 2, E234, 10.1371/journal.pbio.0020234 · DOI
  32. Schroder, 2002, HIV-1 integration in the human genome favors active genes and local hotspots., Cell, 110, 521, 10.1016/S0092-8674(02)00864-4 · DOI · Paper page
  33. Kohn, 2010, Update on gene therapy for immunodeficiencies., Clin Immunol, 135, 247, 10.1016/j.clim.2009.12.003 · DOI
  34. Qasim, 2009, Progress and prospects: gene therapy for inherited immunodeficiencies., Gene Ther, 16, 1285, 10.1038/gt.2009.127 · DOI
  35. Howe, 2008, Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients., J Clin Invest, 118, 3143, 10.1172/JCI35798 · DOI · Paper page
  36. Ryu, 2008, An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation., Blood, 111, 1866, 10.1182/blood-2007-04-085506 · DOI
  37. McCormack, 2010, The Lmo2 oncogene initiates leukemia in mice by inducing thymocyte self-renewal., Science, 327, 879, 10.1126/science.1182378 · DOI
  38. Schmidt, 2003, Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates., Nat Med, 9, 463, 10.1038/nm844 · DOI
  39. Kohn, 2003, Occurrence of leukaemia following gene therapy of X-linked SCID., Nat Rev Cancer, 3, 477, 10.1038/nrc1122 · DOI
  40. Seggewiss, 2006, Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque., Blood, 107, 3865, 10.1182/blood-2005-10-4108 · DOI
  41. Calmels, 2005, Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells., Blood, 106, 2530, 10.1182/blood-2005-03-1115 · DOI
  42. Modlich, 2005, Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis., Blood, 105, 4235, 10.1182/blood-2004-11-4535 · DOI
  43. Kustikova, 2005, Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking., Science, 308, 1171, 10.1126/science.1105063 · DOI · Paper page
  44. Yu, 1986, Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells., Proc Natl Acad Sci U S A, 83, 3194, 10.1073/pnas.83.10.3194 · DOI
  45. Hirata, 1996, Transduction of hematopoietic cells by foamy virus vectors., Blood, 88, 3654, 10.1182/blood.V88.9.3654.bloodjournal8893654 · DOI
  46. Beard, 2007, Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells., Mol Ther, 15, 1356, 10.1038/sj.mt.6300159 · DOI
  47. De Palma, 2005, Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells., Blood, 105, 2307, 10.1182/blood-2004-03-0798 · DOI
  48. Hematti, 2004, Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells., PLoS Biol, 2, e423, 10.1371/journal.pbio.0020423 · DOI
  49. Biffi, 2011, Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection., Blood, 117, 5332, 10.1182/blood-2010-09-306761 · DOI · Paper page
  50. Deichmann, 2007, Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy., J Clin Invest, 117, 2225, 10.1172/JCI31659 · DOI
  51. Deichmann, 2011, Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy., Mol Ther, 19, 2031, 10.1038/mt.2011.178 · DOI
  52. Ciuffi, 2008, Mechanisms governing lentivirus integration site selection., Curr Gene Ther, 8, 419, 10.2174/156652308786848021 · DOI
  53. Bushman, 2005, Genome-wide analysis of retroviral DNA integration., Nat Rev Microbiol, 3, 848, 10.1038/nrmicro1263 · DOI
  54. Lu, 2011, Tracking single hematopoietic stem cells in vivo using high-throughput sequencing in conjunction with viral genetic barcoding., Nat Biotechnol, 29, 928, 10.1038/nbt.1977 · DOI
  55. Brady, 2011, A method to sequence and quantify DNA integration for monitoring outcome in gene therapy., Nucleic Acids Res, 39, e72, 10.1093/nar/gkr140 · DOI
  56. Chang, 2007, The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors., Mol Ther, 15, 445, 10.1038/sj.mt.6300060 · DOI
  57. Modlich, 2009, Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors., Mol Ther, 17, 1919, 10.1038/mt.2009.179 · DOI · Paper page
  58. Anderson, 2007, Safety and efficacy of a lentiviral vector containing three anti-HIV genes–CCR5 ribozyme, tat-rev siRNA, and TAR decoy–in SCID-hu mouse-derived T cells., Mol Ther, 15, 1182, 10.1038/sj.mt.6300157 · DOI
  59. Li, 2005, Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy., Mol Ther, 12, 900, 10.1016/j.ymthe.2005.07.524 · DOI
  60. DiGiusto, 2010, RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma., Sci Transl Med, 2, 36ra43, 10.1126/scitranslmed.3000931 · DOI
  61. Sadelain, 2008, Stem cell engineering for the treatment of severe hemoglobinopathies., Curr Mol Med, 8, 690, 10.2174/156652408786241357 · DOI
  62. Ikeda, 2011, 3′UTR-truncated Hmga2 cDNA causes MPN-like hematopoiesis by conferring a clonal growth advantage at the level of HSC in mice., Blood, 117, 5860, 10.1182/blood-2011-02-334425 · DOI
  63. Giles, 2010, Chromatin boundaries, insulators, and long-range interactions in the nucleus., Cold Spring Harb Symp Quant Biol, 75, 79, 10.1101/sqb.2010.75.006 · DOI
  64. Emery, 2011, The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors., Hum Gene Ther, 22, 761, 10.1089/hum.2010.233 · DOI
  65. Li, 2009, Genomic and functional assays demonstrate reduced gammaretroviral vector genotoxicity associated with use of the cHS4 chromatin insulator., Mol Ther, 17, 716, 10.1038/mt.2009.7 · DOI
  66. Desprat, 2009, Gene specificity of suppression of transgene-mediated insertional transcriptional activation by the chicken HS4 insulator., PLoS One, 4, e5956, 10.1371/journal.pone.0005956 · DOI
  67. Brown, 2009, Exploiting and antagonizing micro-RNA regulation for therapeutic and experimental applications., Nat Rev Genet, 10, 578, 10.1038/nrg2628 · DOI · Paper page
  68. Erlwein, 2010, Progress and prospects: foamy virus vectors enter a new age., Gene Ther, 17, 1423, 10.1038/gt.2010.95 · DOI
  69. Bauer, 2008, Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors., Nat Med, 14, 93, 10.1038/nm1695 · DOI
  70. Hu, 2008, Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors., Mol Ther, 16, 1617, 10.1038/mt.2008.135 · DOI
  71. Preuss, 2010, TK. 007: A novel, codon-optimized HSVtk(A168H) mutant for suicide gene therapy., Hum Gene Ther, 21, 929, 10.1089/hum.2009.042 · DOI
  72. Straathof, 2005, An inducible caspase 9 safety switch for T-cell therapy., Blood, 105, 4247, 10.1182/blood-2004-11-4564 · DOI
  73. Modlich, 2006, Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity., Blood, 108, 2545, 10.1182/blood-2005-08-024976 · DOI · Paper page
  74. Du, 2005, Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells., Blood, 106, 3932, 10.1182/blood-2005-03-1113 · DOI
  75. Zhang, 2008, High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector., J Clin Invest, 118, 1502, 10.1172/JCI34371 · DOI
  76. Porteus, 2005, Gene targeting using zinc finger nucleases., Nat Biotechnol, 23, 967, 10.1038/nbt1125 · DOI · Paper page
  77. Suzuki, 2008, Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors., Proc Natl Acad Sci U S A, 105, 13781, 10.1073/pnas.0806976105 · DOI
  78. Khan, 2010, Engineering of human pluripotent stem cells by AAV-mediated gene targeting., Mol Ther, 18, 1192, 10.1038/mt.2010.55 · DOI
  79. Knauert, 2001, Triplex forming oligonucleotides: sequence-specific tools for gene targeting., Hum Mol Genet, 10, 2243, 10.1093/hmg/10.20.2243 · DOI
  80. McNeer, 2011, Nanoparticles deliver triplex-forming PNAs for site-specific genomic recombination in CD34+ human hematopoietic progenitors., Mol Ther, 19, 172, 10.1038/mt.2010.200 · DOI
  81. Jasin, 1996, Genetic manipulation of genomes with rare-cutting endonucleases., Trends Genet, 12, 224, 10.1016/0168-9525(96)10019-6 · DOI
  82. Paques, 2007, Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy., Curr Gene Ther, 7, 49, 10.2174/156652307779940216 · DOI
  83. Boch, 2011, TALEs of genome targeting., Nat Biotechnol, 29, 135, 10.1038/nbt.1767 · DOI
  84. Holt, 2010, Human hematopoietic stem/progenitor cells modified by zinc finger nucleases targeted to CCR5 control HIV-1 in vivo., Nat Biotechnol, 28, 839, 10.1038/nbt.1663 · DOI · Paper page
  85. Radecke, 2010, Zinc finger nuclease-induced gene repair with oligodeoxynucleotides: wanted and unwanted target locus modifications., Mol Ther, 18, 743, 10.1038/mt.2009.304 · DOI
  86. Gabriel, 2011, An unbiased genome-wide analysis of zinc finger nuclease specificity., Nat Biotechnol, 29, 816, 10.1038/nbt.1948 · DOI · Paper page
  87. Pattanayak, 2011, Revealing off-target cleavage specificities of zinc finger nucleases by in vitro selection., Nat Methods, 8, 765, 10.1038/nmeth.1670 · DOI · Paper page
  88. Takahashi, 2006, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors., Cell, 126, 663, 10.1016/j.cell.2006.07.024 · DOI · Paper page
  89. Hanna, 2007, Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin., Science, 318, 1920, 10.1126/science.1152092 · DOI · Paper page
  90. Yamanaka, 2007, Strategies and new developments in the generation of patient-specific pluripotent stem cells., Cell Stem Cell, 1, 39, 10.1016/j.stem.2007.05.012 · DOI
  91. Kane, 2010, Lentivirus-mediated reprogramming of somatic cells in the absence of transgenic transcription factors., Mol Ther, 18, 2139, 10.1038/mt.2010.231 · DOI
  92. Zou, 2009, Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells., Cell Stem Cell, 5, 97, 10.1016/j.stem.2009.05.023 · DOI
  93. Song, 2010, Modeling disease in human ESCs using an efficient BAC-based homologous recombination system., Cell Stem Cell, 6, 80, 10.1016/j.stem.2009.11.016 · DOI
  94. Papapetrou, 2011, Genomic safe harbors permit high beta-globin transgene expression in thalassemia induced pluripotent stem cells., Nat Biotechnol, 29, 73, 10.1038/nbt.1717 · DOI
  95. Sadelain, Safe harbours for the integration of new DNA in the human genome [published online ahead of print December 1, 2011]., Nat Rev Cancer
  96. Hussein, 2011, Copy number variation and selection during reprogramming to pluripotency., Nature, 471, 58, 10.1038/nature09871 · DOI
  97. Li, 2009, The Ink4/Arf locus is a barrier for iPS cell reprogramming., Nature, 460, 1136, 10.1038/nature08290 · DOI · Paper page
  98. Draper, 2004, Culture and characterization of human embryonic stem cells., Stem Cells Dev, 13, 325, 10.1089/scd.2004.13.325 · DOI
  99. Baker, 2007, Adaptation to culture of human embryonic stem cells and oncogenesis in vivo., Nat Biotechnol, 25, 207, 10.1038/nbt1285 · DOI
  100. Mayshar, 2010, Identification and classification of chromosomal aberrations in human induced pluripotent stem cells., Cell Stem Cell, 7, 521, 10.1016/j.stem.2010.07.017 · DOI · Paper page
  101. Howden, 2011, Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy., Proc Natl Acad Sci U S A, 108, 6537, 10.1073/pnas.1103388108 · DOI
  102. Zhao, 2011, Immunogenicity of induced pluripotent stem cells., Nature, 474, 212, 10.1038/nature10135 · DOI
  103. Robert-Moreno, 2008, Impaired embryonic haematopoiesis yet normal arterial development in the absence of the Notch ligand Jagged1., EMBO J, 27, 1886, 10.1038/emboj.2008.113 · DOI
  104. Eilken, 2009, Continuous single-cell imaging of blood generation from haemogenic endothelium., Nature, 457, 896, 10.1038/nature07760 · DOI
  105. Zovein, 2008, Fate tracing reveals the endothelial origin of hematopoietic stem cells., Cell Stem Cell, 3, 625, 10.1016/j.stem.2008.09.018 · DOI · Paper page
  106. Rhodes, 2008, The emergence of hematopoietic stem cells is initiated in the placental vasculature in the absence of circulation., Cell Stem Cell, 2, 252, 10.1016/j.stem.2008.01.001 · DOI
  107. Kissa, 2010, Blood stem cells emerge from aortic endothelium by a novel type of cell transition., Nature, 464, 112, 10.1038/nature08761 · DOI
  108. Boisset, 2010, In vivo imaging of haematopoietic cells emerging from the mouse aortic endothelium., Nature, 464, 116, 10.1038/nature08764 · DOI · Paper page
  109. Wang, 2005, Generation of hematopoietic repopulating cells from human embryonic stem cells independent of ectopic HOXB4 expression., J Exp Med, 201, 1603, 10.1084/jem.20041888 · DOI
  110. Raya, 2009, Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells., Nature, 460, 53, 10.1038/nature08129 · DOI
  111. Tian, 2009, Bioluminescent imaging demonstrates that transplanted human embryonic stem cell-derived CD34(+) cells preferentially develop into endothelial cells., Stem Cells, 27, 2675, 10.1002/stem.204 · DOI
  112. Ledran, 2008, Efficient hematopoietic differentiation of human embryonic stem cells on stromal cells derived from hematopoietic niches., Cell Stem Cell, 3, 85, 10.1016/j.stem.2008.06.001 · DOI
  113. Rideout, 2002, Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy., Cell, 109, 17, 10.1016/S0092-8674(02)00681-5 · DOI
  114. Schiedlmeier, 2003, High-level ectopic HOXB4 expression confers a profound in vivo competitive growth advantage on human cord blood CD34+ cells but impairs lymphomyeloid differentiation., Blood, 101, 1759, 10.1182/blood-2002-03-0767 · DOI
  115. Zhang, 2007, Effects of HOXB4 overexpression on ex vivo expansion and immortalization of hematopoietic cells from different species., Stem Cells, 25, 2074, 10.1634/stemcells.2006-0742 · DOI
  116. Szabo, 2010, Direct conversion of human fibroblasts to multilineage blood progenitors., Nature, 468, 521, 10.1038/nature09591 · DOI
  117. Graf, 2009, Forcing cells to change lineages., Nature, 462, 587, 10.1038/nature08533 · DOI · Paper page
  118. Trobridge, 2006, Foamy virus vector integration sites in normal human cells., Proc Natl Acad Sci U S A, 103, 1498, 10.1073/pnas.0510046103 · DOI
  119. Sadelain, 2011, Eliminating cells gone astray., N Engl J Med, 365, 1735, 10.1056/NEJMe1109971 · DOI
  120. Hacein-Bey, 1996, gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function., Blood, 87, 3108, 10.1182/blood.V87.8.3108.bloodjournal8783108 · DOI
  121. Hacein-Bey-Abina, 2003, LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1., Science, 302, 415, 10.1126/science.1088547 · DOI · Paper page
  122. Gaspar, 2004, Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector., Lancet, 364, 2181, 10.1016/S0140-6736(04)17590-9 · DOI
  123. Imren, 2002, Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells., Proc Natl Acad Sci U S A, 99, 14380, 10.1073/pnas.212507099 · DOI

Cited by

Metadata

Authors
Rivière, I., Dunbar, C. E., & Sadelain, M.
Affiliations
  • Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan-Kettering Cancer Center, New York, NY; and
  • Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
Venue
Blood
Publisher
American Society of Hematology
Published
2012-02-02
Volume
119
Issue
5
Pages
1107-1116
ISSN
  • 0006-4971
  • 1528-0020
DOI
10.1182/blood-2011-09-349993
Cited by
54
References
18
Language
en

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review