C E Dunbar

Summary

Affiliation: National Institutes of Health
Country: USA

Publications

  1. pmc Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
    Boris Calmels
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    Blood 106:2530-3. 2005
  2. ncbi request reprint Stem cell gene transfer: insights into integration and hematopoiesis from primate genetic marking studies
    Cynthia E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10 CRC, Room 4E 5132, Bethesda, MD 20892 1202, USA
    Ann N Y Acad Sci 1044:178-82. 2005
  3. ncbi request reprint Genotoxicity of retroviral integration in hematopoietic cells
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, TN 38103, USA
    Mol Ther 13:1031-49. 2006
  4. pmc Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors
    Jingqiong Hu
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, Blood Institute, National Institute of Health, Bethesda, Maryland, USA
    Mol Ther 16:1617-23. 2008
  5. pmc BCL2A1a over-expression in murine hematopoietic stem and progenitor cells decreases apoptosis and results in hematopoietic transformation
    Jean Yves Métais
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States of America
    PLoS ONE 7:e48267. 2012
  6. pmc Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
    Peiman Hematti
    Hematology Branch, National Institutes of Health Bethesda, Maryland, USA
    PLoS Biol 2:e423. 2004
  7. ncbi request reprint The impact of ex vivo cytokine stimulation on engraftment of primitive hematopoietic cells in a non-human primate model
    C E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch, NHLBI, NIH, Building 10, Room 7C103, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Ann N Y Acad Sci 938:236-44; discussion 244-5. 2001
  8. pmc Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor
    C E Dunbar
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Proc Natl Acad Sci U S A 93:11871-6. 1996
  9. pmc Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates
    M Takatoku
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    J Clin Invest 108:447-55. 2001
  10. ncbi request reprint Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability
    J F Tisdale
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Blood 92:1131-41. 1998

Detail Information

Publications82

  1. pmc Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
    Boris Calmels
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    Blood 106:2530-3. 2005
    ..Characterization of integration sites in this relevant preclinical model provides critical information for gene therapy risk assessment as well as identification of genes controlling hematopoiesis...
  2. ncbi request reprint Stem cell gene transfer: insights into integration and hematopoiesis from primate genetic marking studies
    Cynthia E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10 CRC, Room 4E 5132, Bethesda, MD 20892 1202, USA
    Ann N Y Acad Sci 1044:178-82. 2005
    ....
  3. ncbi request reprint Genotoxicity of retroviral integration in hematopoietic cells
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, TN 38103, USA
    Mol Ther 13:1031-49. 2006
    ..The future of such efforts seems bright as we continue to evolve and improve various strategies to make such interventions both effective and as safe as possible...
  4. pmc Reduced genotoxicity of avian sarcoma leukosis virus vectors in rhesus long-term repopulating cells compared to standard murine retrovirus vectors
    Jingqiong Hu
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, Blood Institute, National Institute of Health, Bethesda, Maryland, USA
    Mol Ther 16:1617-23. 2008
    ..The combination of these features is unique for ASLV and suggests that optimized vectors based on this virus could be useful and safe for gene transfer to hematopoietic stem cells and progenitor cells...
  5. pmc BCL2A1a over-expression in murine hematopoietic stem and progenitor cells decreases apoptosis and results in hematopoietic transformation
    Jean Yves Métais
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, United States of America
    PLoS ONE 7:e48267. 2012
    ....
  6. pmc Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
    Peiman Hematti
    Hematology Branch, National Institutes of Health Bethesda, Maryland, USA
    PLoS Biol 2:e423. 2004
    ..These integration patterns suggest different mechanisms for integration as well as distinct safety implications for MLV versus SIV vectors...
  7. ncbi request reprint The impact of ex vivo cytokine stimulation on engraftment of primitive hematopoietic cells in a non-human primate model
    C E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch, NHLBI, NIH, Building 10, Room 7C103, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Ann N Y Acad Sci 938:236-44; discussion 244-5. 2001
    ..This approach may allow more efficient engraftment of successfully transduced or ex vivo expanded cells by avoiding active cell cycling at the time of reinfusion...
  8. pmc Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor
    C E Dunbar
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Proc Natl Acad Sci U S A 93:11871-6. 1996
    ....
  9. pmc Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates
    M Takatoku
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    J Clin Invest 108:447-55. 2001
    ..We observed stable in vivo overall gene marking levels of up to 29%. This approach may allow more efficient engraftment of transduced or ex vivo expanded cells by avoiding active cell cycling at the time of reinfusion...
  10. ncbi request reprint Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability
    J F Tisdale
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Blood 92:1131-41. 1998
    ..However, a brief transduction in the presence of specific cytokines and stromal support allows engraftment with an encouraging number of retrovirally modified cells...
  11. pmc Evaluation of a rapamycin-regulated serotype 2 adeno-associated viral vector in macaque parotid glands
    C Zheng
    Molecular Physiology and Therapeutics Branch, National Institute of Dental and Craniofacial Research, Bethesda, MD 20892 1190, USA
    Oral Dis 16:269-77. 2010
    ..This study evaluated if such a vector was similarly useful in rhesus macaque parotid glands...
  12. ncbi request reprint In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting
    E Kang
    Molecular and Clinical Hematology Branch, National Institute of Diabetes and Digestive and Kidney Disorders/NIH, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Hum Gene Ther 12:1663-72. 2001
    ..We conclude that even with very low dose conditioning, engraftment by genetically modified LTRC cells at clinically significant levels can be achieved without evidence for clearance of cells known to be expressing immunogenic proteins...
  13. ncbi request reprint The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells
    S E Sellers
    Hematology Branch, The National Heart, Lung, and Blood Institute, and the Molecular and Clinical Hematology Branch, The National Institute of Diabetes and Digestive and Kidney Diseases, The National Institutes of Health, Bethesda, MD 20892, USA
    Blood 97:1888-91. 2001
    ..There was no evidence for expansion of MDR1 vector-transduced cells. Long-term engraftment with MDR1 vector- and neo vector-transduced cells occurred despite prolonged culture...
  14. ncbi request reprint Assessment of rapid remobilization intervals with G-CSF and SCF in murine and rhesus macaque models
    P A Shi
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892 1652, USA
    Transfusion 41:1438-44. 2001
    ..Defining the optimum regimen and time for repeat peripheral blood progenitor cell mobilization would have important clinical applications...
  15. ncbi request reprint The use of nonhuman primate models to improve gene transfer into haematopoietic stem cells
    C E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Rockville Pike, Bethesda, MD, USA
    J Intern Med 249:329-38. 2001
    ..These and other advances should result in successful gene therapy for a variety of acquired and congenital disorders affecting HSCs and their progeny lineages...
  16. ncbi request reprint Analysis of origin and optimization of expansion and transduction of circulating peripheral blood endothelial progenitor cells in the rhesus macaque model
    J Hu
    Hematology Branch, National Heart, Lung, and Blood Institute NIH, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Hum Gene Ther 13:2041-50. 2002
    ..Further elucidation of the origin and in vivo behavior of EPCs may be possible, using optimized transduction conditions and a vascular injury model...
  17. ncbi request reprint Update on the use of nonhuman primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells
    R E Donahue
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD 20892, USA
    Hum Gene Ther 12:607-17. 2001
    ..Judicious application of these models should continue to be a priority, and advances should now be tested in proof-of-concept clinical trials...
  18. ncbi request reprint In vivo gene marking of rhesus macaque long-term repopulating hematopoietic cells using a VSV-G pseudotyped versus amphotropic oncoretroviral vector
    Patricia A Shi
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institues of Health, Bethesda, MD 20892, USA
    J Gene Med 6:367-73. 2004
    ..Vectors pseudotyped with the vesicular stomatitis virus (VSV-G) envelope do not need receptors to enter cells, and therefore may provide superior transduction efficiency...
  19. pmc Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells
    Yoo Jin Kim
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health NIH, Bethesda, MD 20852, USA
    Blood 113:5434-43. 2009
    ....
  20. ncbi request reprint Transplant dose of CD34(+) and CD3(+) cells predicts outcome in patients with haematological malignancies undergoing T cell-depleted peripheral blood stem cell transplants with delayed donor lymphocyte add-back
    R Nakamura
    Stem Cell Transplantation Unit, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Br J Haematol 115:95-104. 2001
    ..83 x 10(5) CD3(+) cells/kg. These results further define the impact of CD34 and CD3 cell dose on transplant outcome and show that careful dosing of stem cells and lymphocytes may permit the control and optimization of transplant outcome...
  21. pmc AAV5-mediated gene transfer to the parotid glands of non-human primates
    A Voutetakis
    Molecular Physiology and Therapeutics Branch, National Institute of Dental and Craniofacial Research, Bethesda, MD 20892 1190, USA
    Gene Ther 17:50-60. 2010
    ..The aggregate data indicate that results with AAV5 vectors in murine salivary glands apparently do not extend to macaque glands...
  22. pmc Feline leukemia virus integrase and capsid packaging functions do not change the insertion profile of standard Moloney retroviral vectors
    J Y Métais
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Gene Ther 17:799-804. 2010
    ..However, considering the stability and efficacy of CatPac vectors, further development is warranted, using potentially safer vector backbones, for instance those with a SIN configuration...
  23. pmc AMD3100 mobilizes hematopoietic stem cells with long-term repopulating capacity in nonhuman primates
    Andre Larochelle
    National Heart, Lung, and Blood Institute, National Institutes of Health, Bldg 10 CRC, Rm 4E 5132, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Blood 107:3772-8. 2006
    ..Thus, AMD3100-mobilized CD34(+) cells represent an alternative source of hematopoietic stem cells for clinical stem cell transplantation and genetic manipulation with integrating retroviral vectors...
  24. pmc Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate
    Jianjun Xie
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hum Gene Ther 21:695-703. 2010
    ....
  25. ncbi request reprint Retroviral transduction efficiency of G-CSF+SCF-mobilized peripheral blood CD34+ cells is superior to G-CSF or G-CSF+Flt3-L-mobilized cells in nonhuman primates
    Peiman Hematti
    Hematology Branch, NHLBI, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 101:2199-205. 2003
    ..The difference in transduction efficiency of HSCs collected from different sources should be considered whenever planning clinical gene therapy trials and should preferably be tested directly in comparative studies...
  26. ncbi request reprint Rabaptin-5 is a novel fusion partner to platelet-derived growth factor beta receptor in chronic myelomonocytic leukemia
    M K Magnusson
    Hematology Branch, National Heart, Lung, and Blood Institute, and Laboratory of Pathology, Division of Clinical Sciences, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 98:2518-25. 2001
    ..Early endosomal transport is critical in regulation of various growth factor receptors, through ligand-induced clathrin-mediated endocytosis, and thus this new fusion protein links together 2 important pathways of growth regulation...
  27. ncbi request reprint The impact of low-dose busulfan on clonal dynamics in nonhuman primates
    Ken Kuramoto
    Molecular Hematopoiesis Section, Hematology Branch, Natiomal Heart, Lung and Blood Institute NIH, Bldg 10, Rm 7C103, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Blood 104:1273-80. 2004
    ..period of suppression of many clones suggests that transplanted HSCs may have a marked competitive advantage if they can engraft and proliferate during this time period, and supports the use of this agent in nonmyeloablative regimens..
  28. pmc HOXB4 and retroviral vectors: adding fuel to the fire
    Andre Larochelle
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    J Clin Invest 118:1350-3. 2008
    ..These results highlight the risks of combining integrating vectors and growth-promoting genes for clinical applications...
  29. ncbi request reprint Genetic marking as an approach to studying in vivo hematopoiesis: progress in the non-human primate model
    Patricia A Shi
    Molecular Hematopoiesis Section, Hematology Branch, NHLBI, NIH, 9000 Rockville Pike, Bethesda, Maryland, MD 20892, USA
    Oncogene 21:3274-83. 2002
    ..These approaches will have significant impact in studying various aspects of stem cell biology including the phenomenon of stem cell plasticity...
  30. pmc MRI detection of single particles for cellular imaging
    Erik M Shapiro
    Laboratory of Functional and Molecular Imaging, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Proc Natl Acad Sci U S A 101:10901-6. 2004
    ..These results demonstrate that MRI can detect single particles and indicate that single-particle detection will be useful for cellular imaging...
  31. pmc Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy
    Thomas R Bauer
    Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, 10 Center Dr, MSC1203, Bldg 10 CRC, Rm 3 3264, Bethesda, MD 20892 1203, USA
    Blood 108:3313-20. 2006
    ....
  32. pmc In vitro culture during retroviral transduction improves thymic repopulation and output after total body irradiation and autologous peripheral blood progenitor cell transplantation in rhesus macaques
    Karin Lore
    Immunology Laboratory, Research Center, National Institute of Allergy and Infectious Diseases, NIH, Department of Health and Human Services, Bethesda, Maryland 20892 1202, USA
    Stem Cells 24:1539-48. 2006
    ..These results have implications for the design of gene therapy trials, as well as for the use of expanded PBPCs for improved T-cell immune reconstitution after transplantation...
  33. ncbi request reprint Genetic manipulation of hematopoietic stem cells
    Andre Larochelle
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Semin Hematol 41:257-71. 2004
    ....
  34. ncbi request reprint Resetting the immune system in immune thrombocytopenic purpura: immunoablative strategies
    Patrick F Fogarty
    Department of Laboratory Medicine, Hematology Service, Warren Grant Magnuson Clinical Center, National Institutes of Health, Bethesda, MD 20892 1508, USA
    Clin Adv Hematol Oncol 1:365-71. 2003
    ..Approaches that will be discussed include conventional chemotherapy, high-dose chemotherapy with and without autologous stem cell support, and allogeneic transplantation...
  35. pmc Ex vivo expansion of retrovirally transduced primate CD34+ cells results in overrepresentation of clones with MDS1/EVI1 insertion sites in the myeloid lineage after transplantation
    Stephanie Sellers
    Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, Maryland 20892 1290, USA
    Mol Ther 18:1633-9. 2010
    ..There was no overrepresentation of MDS1/EVI1 insertions contributing to lymphoid lineages. Strategies involving prolonged ex vivo expansion of transduced cells may increase the risk of genotoxicity...
  36. pmc Keratinocyte growth factor augments immune reconstitution after autologous hematopoietic progenitor cell transplantation in rhesus macaques
    Ruth Seggewiss
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, MD 20892, USA
    Blood 110:441-9. 2007
    ..Thus, our findings suggest that KGF may be a useful adjuvant therapy to augment T-cell reconstitution after human PBPC transplantation...
  37. ncbi request reprint Update on hematopoietic stem cell gene transfer using non-human primate models
    Jiong Hu
    Molecular Hematopoiesis Hematology Branch, National Heart Lung and Blood Institutes, National Institutes of Health, Bethesda, MD 20892, USA
    Curr Opin Mol Ther 4:482-90. 2002
    ..Further development of post-transduction selection and/or expansion strategies using drug-resistance or amplifier genes will most likely be necessary for clinical applications...
  38. ncbi request reprint Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial
    Linda Mesler Muul
    Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 101:2563-9. 2003
    ..This long-term follow-up has also provided unique evidence supporting the safety of retroviral-mediated gene transfer and illustrates clear examples of both the potential and the pitfalls of gene therapy in humans...
  39. ncbi request reprint Activity of STI571 in chronic myelomonocytic leukemia with a platelet-derived growth factor beta receptor fusion oncogene
    Magnus K Magnusson
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 100:1088-91. 2002
    ..These results suggest that STI571 may be an effective targeted therapy in patients with CMML related to PDGFbetaR fusion oncogenes...
  40. ncbi request reprint Plasticity and hematopoiesis: Circe's transforming potion?
    John F Tisdale
    Molecular and Clinical Hematology Branch, National Institute of Diabetes, Digestive and Kidney Diseases, Bethesda, Maryland 20892, USA
    Curr Opin Hematol 9:268-73. 2002
    ..Large animal models should prove invaluable to the progress of the field...
  41. pmc In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene
    Andre Larochelle
    National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    J Clin Invest 119:1952-63. 2009
    ..Our data therefore suggest that lentivirally mediated gene transfer in transplanted HSCs can provide in vivo chemoprotection of progenitor cells, although selection of long-term repopulating HSCs was not seen...
  42. ncbi request reprint Direct comparison of RD114-pseudotyped versus amphotropic-pseudotyped retroviral vectors for transduction of rhesus macaque long-term repopulating cells
    Jiong Hu
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, 9000 Rockville Pike, Bethesda, Maryland 20892, USA
    Mol Ther 8:611-7. 2003
    ..o.i. or inducing the receptor could potentially improve results with this novel vector system...
  43. ncbi request reprint Retroviral transduction and engraftment ability of primate hematopoietic progenitor and stem cells transduced under serum-free versus serum-containing conditions
    Kimberley A Kluge
    Hematology Branch, NHLBI, NIH, Bethesda, Maryland 20892, USA
    Mol Ther 5:316-22. 2002
    ..These data are encouraging regarding the removal of serum-containing medium from clinical hematopoietic cell transduction protocols, given the lack of a detrimental effect on transduction and engraftment with transduced cells...
  44. pmc Relapse following discontinuation of imatinib mesylate therapy for FIP1L1/PDGFRA-positive chronic eosinophilic leukemia: implications for optimal dosing
    Amy D Klion
    Laboratory of Parasitic Diseases, National Institutes of Allergy and Infectious Diseases, National Institutes of Health NIH, Bethesda, MD 20892, USA
    Blood 110:3552-6. 2007
    ..This trial was registered at http://www.clinicaltrials.gov as no. NCT00044304...
  45. ncbi request reprint Stem and progenitor cell therapies: insights from non-human primate models
    C E Dunbar
    Molecular Hematopoiesis Section, Hematology Branch National Heart, Lung, and Blood Institute, NIH, Bethesda, Maryland, USA
    Cytotherapy 6:586-8. 2004
    ....
  46. ncbi request reprint High-dose cyclophosphamide in severe aplastic anaemia: a randomised trial
    J F Tisdale
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Lancet 356:1554-9. 2000
    ....
  47. ncbi request reprint Transduction of rhesus macaque hematopoietic stem and progenitor cells with avian sarcoma and leukosis virus vectors
    Jingqiong Hu
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hum Gene Ther 18:691-700. 2007
    ..These findings are encouraging and suggest that this vector system should be explored and further optimized for gene therapy applications targeting hematopoietic stem and progenitor cells...
  48. ncbi request reprint Absence of donor-derived keratinocyte stem cells in skin tissues cultured from patients after mobilized peripheral blood hematopoietic stem cell transplantation
    Peiman Hematti
    Hematology Branch, NHLBI, National Institutes of Health, Bethesda, MD 20892, USA
    Exp Hematol 30:943-9. 2002
    ..We investigated the contribution of cells with a primitive hematopoietic phenotype to human epidermal skin formation in recipients of allogeneic mobilized peripheral blood hematopoietic stem cell (HSC) transplantation...
  49. ncbi request reprint Effect of chronic cytokine therapy on clonal dynamics in nonhuman primates
    Ken Kuramoto
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 103:4070-7. 2004
    ..These relevant large animal studies are reassuring regarding clinical applications of cytokines and provide new insights into their mechanisms of action...
  50. doi request reprint Active thrombopoiesis is associated with worse severity and activity of chronic GVHD
    T Bat
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Bone Marrow Transplant 48:1569-73. 2013
    ..Among patients with stable moderate or severe cGVHD, there was no evidence of hypoproduction of platelets. Future studies should further investigate the role of thrombopoiesis in cGVHD. ..
  51. ncbi request reprint BM-derived cell therapies for cardiovascular disease
    R O Cannon
    Cardiology Branch and Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    Cytotherapy 9:305-15. 2007
  52. ncbi request reprint A pilot study of nonmyeloablative allogeneic hematopoietic stem cell transplant for advanced systemic mastocytosis
    R Nakamura
    Stem Cell Allogeneic Transplant Unit, Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD, USA
    Bone Marrow Transplant 37:353-8. 2006
    ..The GvMC effect can be observed after nonmyeloablative HCT with limited efficacy. Effective cytoreductive therapy prior to HCT may be required for long-term disease control and cure...
  53. ncbi request reprint Highly efficient endosomal labeling of progenitor and stem cells with large magnetic particles allows magnetic resonance imaging of single cells
    Kathleen A Hinds
    Hematology Branch, Laboratory of Molecular Biology, Laboratory National Heart, Lung, and Blood Institute NHLBI, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 102:867-72. 2003
    ..MRI studies could detect labeled CD34+ cells and mesenchymal stem cells (MSCs) at single cell resolution. This appears to be a promising tool for serial noninvasive monitoring of in vivo cell homing and localization using MRI...
  54. ncbi request reprint The presence of the carboxy-terminal fragment of fibronectin allows maintenance of non-human primate long-term hematopoietic repopulating cells during extended ex vivo culture and transduction
    Stephanie E Sellers
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, 20892, USA
    Exp Hematol 32:163-70. 2004
    ....
  55. pmc A new direction for gene therapy: intrathymic T cell-specific lentiviral gene transfer
    Ruth Seggewiss
    National Heart, Lung, and Blood Institute, NIH, Bethesda, Maryland 20892, USA
    J Clin Invest 115:2064-7. 2005
    ....
  56. doi request reprint The MDS1-EVI1 gene complex as a retrovirus integration site: impact on behavior of hematopoietic cells and implications for gene therapy
    Jean Yves Métais
    Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
    Mol Ther 16:439-49. 2008
    ..Insights into the mechanisms that result in altered hematopoiesis and leukemogenesis when this locus is dysregulated could improve the safety of gene therapy in the future...
  57. ncbi request reprint Adeno-associated virus serotype 2-mediated gene transfer to the parotid glands of nonhuman primates
    Antonis Voutetakis
    Gene Therapy and Therapeutics Branch, National Institute of Dental and Craniofacial Research, Department of Human Health Services, Bethesda, MD 20892, USA
    Hum Gene Ther 18:142-50. 2007
    ..We conclude that administration of modest doses of rAAV2 vectors to SGs for therapeutic purposes can be accomplished without significant or permanent injury to the targeted gland or to distant organs of nonhuman primates...
  58. ncbi request reprint Low-dose total body irradiation causes clonal fluctuation of primate hematopoietic stem and progenitor cells
    Mikko O Laukkanen
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bldg 10, Rm 7C103, 9000 Rockville Pike, Bethesda, MD 20892, USA
    Blood 105:1010-5. 2005
    ..The results indicate that even low-dose irradiation affects hematopoietic clonal dynamics and have implications for design of conditioning regimens for transplantation purposes...
  59. ncbi request reprint Hematopoietic stem cell gene therapy: dead or alive?
    Cole Ferguson
    Molecular Hematopoiesis Section, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Trends Biotechnol 23:589-97. 2005
    ..Scientists are continually revising protocols: going both from "bench to bedside" and, as strikingly demonstrated by HSC gene therapy, from "bedside to bench."..
  60. doi request reprint Large animal models for stem and progenitor cell analysis
    Robert E Donahue
    National Institutes of Health, Bethesda, Maryland, USA
    Curr Protoc Immunol . 2005
    ....
  61. pmc Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque
    Ruth Seggewiss
    Hematology Branch, National Heart, Lung, and Blood Institute, NIH DHHS, Bldg 10, CRC, Rm 4 5130, 10 Center Drive, MSC 1202, Bethesda, MD 20892, USA
    Blood 107:3865-7. 2006
    ..One insertion was found in BCL2-A1, an antiapoptotic gene. This event suggests that currently available retroviral vectors may have long-term side effects, particularly in hematopoietic stem and progenitor cells...
  62. pmc T cell receptor VB repertoire diversity in patients with immune thrombocytopenia following splenectomy
    P F Fogarty
    Hematology Section, Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892 1652, USA
    Clin Exp Immunol 133:461-6. 2003
    ..These findings suggest that removal of the spleen may lead directly or indirectly to reductions in T cell clonal expansions in responders, or that the extent of T cell clonality impacts responsiveness to splenectomy in patients with ITP...
  63. ncbi request reprint Cyclosporine is required to prevent severe acute GVHD following T-cell-depleted peripheral blood stem cell transplantation
    S R Solomon
    Stem Cell Allotransplantation Section, Hematology Branch, NHLBI, National Institutes of Health, Bathesda, MD 20892, USA
    Bone Marrow Transplant 31:783-8. 2003
    ..Similarly, no significant differences were found in chronic GVHD, transplant-related mortality, or survival. These results define a role for CSA in preventing GVHD at low T-cell doses following PBSCT...
  64. pmc Sorting of transgenic secretory proteins in rhesus macaque parotid glands after adenovirus-mediated gene transfer
    Antonis Voutetakis
    Molecular Physiology and Therapeutics Branch, National Institute of Dental and Craniofacial Research, Department of Health and Human Services, Bethesda, MD 20892, USA
    Hum Gene Ther 19:1401-5. 2008
    ..We conclude that RSP proteins are faithfully secreted into saliva in all model species tested, whereas patterns of CSP protein secretion are variable...
  65. pmc No evidence for clonal selection due to lentiviral integration sites in human induced pluripotent stem cells
    Thomas Winkler
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, Maryland, USA
    Stem Cells 28:687-94. 2010
    ..Our study supports recent reports that efficient reprogramming of human somatic cells is not dependent on insertional activation or deactivation of specific genes or gene classes...
  66. ncbi request reprint Imatinib inhibits T-cell receptor-mediated T-cell proliferation and activation in a dose-dependent manner
    Ruth Seggewiss
    Hematology Branch, National Heart, Lung, and Blood Institute NHLBI, the Immunology Laboratory, NIH DHHS, Bldg 10, CRC, Rm 4 5140, 10 Center Dr, MSC 1202, Bethesda, MD 20892, USA
    Blood 105:2473-9. 2005
    ....
  67. pmc Serial cardiac magnetic resonance imaging of injected mesenchymal stem cells
    Jonathan M Hill
    Cardiovascular Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Building 10, Room 2c713, Bethesda, MD 20892 1538, USA
    Circulation 108:1009-14. 2003
    ..We hypothesized that mesenchymal stem cells (MSCs) could be labeled with a iron fluorophore particle (IFP) to provide MRI contrast in vivo to assess immediate and long-term localization...
  68. ncbi request reprint High-dose cyclophosphamide with autologous lymphocyte-depleted peripheral blood stem cell (PBSC) support for treatment of refractory chronic autoimmune thrombocytopenia
    Richard D Huhn
    Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
    Blood 101:71-7. 2003
    ..This therapeutic approach is feasible for patients with severe chronic AITP, a substantial proportion of whom may obtain durable remissions. Larger controlled trials are recommended...
  69. pmc Donor demographic and laboratory predictors of allogeneic peripheral blood stem cell mobilization in an ethnically diverse population
    Sumithira Vasu
    Department of Transfusion Medicine, Warren Grant Magnuson Clinical Center, National Institute of Diabetes, Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 112:2092-100. 2008
    ..Age, white ethnicity, and female gender were associated with significantly lower post-G-CSF CD34(+) cell counts...
  70. pmc Intercellular transfer to signalling endosomes regulates an ex vivo bone marrow niche
    Jennifer M Gillette
    Cell Biology and Metabolism Program, National Institute of Child Health and Human Development, National Institutes of Health, 9000 Rockville Pike, Bethesda, Maryland 20892, USA
    Nat Cell Biol 11:303-11. 2009
    ..These findings identify a mechanism involving intercellular transfer to signalling endosomes for targeted regulation of signalling and remodelling events within an ex vivo osteoblastic niche...
  71. pmc Cytokine-independent growth and clonal expansion of a primary human CD8+ T-cell clone following retroviral transduction with the IL-15 gene
    Cary Hsu
    Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Blood 109:5168-77. 2007
    ..It does not appear that the retroviral vector integration sites played a role in the continuous growth of this cell clone, but this remains under investigation...
  72. ncbi request reprint Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia
    Hans Peter Kiem
    Clinical Research Division, Fred Hutchinson Cancer Research Center, and Division of Orcology, University of Washington, Seattle, WA, USA
    Mol Ther 9:389-95. 2004
    ....
  73. pmc Mobilization as a preparative regimen for hematopoietic stem cell transplantation
    Jing Chen
    Medicine Hematology, University of Washington, Box 357710, Seattle, 98195, USA
    Blood 107:3764-71. 2006
    ..These results indicate that the number of available niches regulates the number of HSCs. In addition, mobilization with AMD3100 may provide a safer preparative approach for HSC transplantation in genetic and other nonmalignant disorders...
  74. ncbi request reprint Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vector
    Patrick F Kelly
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood Cells Mol Dis 30:132-43. 2003
    ..Our studies provide insights into the biology of hematopoietic reconstitution and suggest approaches for increasing stem cell targeted gene transfer efficiency...
  75. ncbi request reprint Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors
    Derek A Persons
    Department of Hematology Oncology, Division of Experimental Hematology, St Jude Children s Research Hospital, 332 N Lauderdale, Memphis, TN 38105, USA
    Blood 103:796-803. 2004
    ....
  76. pmc Hematopoietic stem-cell behavior in nonhuman primates
    Bryan E Shepherd
    Department of Biostatistics, Vanderbilt University, Nashville, TN, USA
    Blood 110:1806-13. 2007
    ..Thus, our data suggest that the self-renewal capacity of mammalian stem cells in vivo is defined and evolutionarily conserved...
  77. ncbi request reprint Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing
    Manfred Schmidt
    Department I of Internal Medicine, University of Freiburg, 79106 Freiburg, Germany
    Ann N Y Acad Sci 996:112-21. 2003
    ....
  78. ncbi request reprint No evidence of clonal dominance in primates up to 4 years following transplantation of multidrug resistance 1 retrovirally transduced long-term repopulating cells
    Farastuk Bozorgmehr
    Research Group Pharmacology of Cancer Treatment, German Cancer Research Center, Heidelberg, Germany
    Stem Cells 25:2610-8. 2007
    ..0008% +/- 0.0003% to 0.0041% +/- 0.00032% of primate peripheral blood cells. No clonal dominance was observed. Disclosure of potential conflicts of interest is found at the end of this article...
  79. ncbi request reprint Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system
    Hideki Hanawa
    Experimental Hematology Division, Department of Hematology Oncology, St Jude Children s Research Hospital, 332 N Lauderdale, Memphis, TN 38105, USA
    Blood 103:4062-9. 2004
    ..Vector insertion site analysis demonstrated polyclonal reconstitution with vector-containing cells. SIV vectors appear promising for evaluating gene therapy approaches in nonhuman primate models...
  80. ncbi request reprint Antibody-mediated cell labeling of peripheral T cells with micron-sized iron oxide particles (MPIOs) allows single cell detection by MRI
    Erik M Shapiro
    Molecular and Cellular Imaging Laboratory, Department of Diagnostic Radiology, Yale University School of Medicine, New Haven, CT, USA
    Contrast Media Mol Imaging 2:147-53. 2007
    ..Therefore, the use of MPIOs for achieving high iron concentrations for cellular MRI is potentially an effective new modality for non-invasive imaging of lymphocytes...
  81. ncbi request reprint Gene therapy for hematologic disease: Don't throw the baby out with the bathwater!
    Cynthia E Dunbar
    Semin Hematol 41:1-2. 2004
  82. ncbi request reprint Polyclonal long-term repopulating stem cell clones in a primate model
    Manfred Schmidt
    Department I of Internal Medicine and the Institute for Molecular Medicine and Cell Research, University of Freiburg, Germany
    Blood 100:2737-43. 2002
    ..Our study provides direct molecular evidence for a polyclonal, multilineage, and sustained contribution of individual stem cells to primate hematopoiesis...