Arthur W Nienhuis

Summary

Affiliation: St. Jude Children's Research Hospital
Country: USA

Publications

  1. ncbi request reprint Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood
    Hideki Hanawa
    Department of Hematology Oncology, Division of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Mol Ther 5:242-51. 2002
  2. ncbi request reprint Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia
    Hideki Hanawa
    332 North Lauderdale Dr, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 104:2281-90. 2004
  3. ncbi request reprint Development of gene therapy for hemoglobin disorders
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, Tennessee 38105, USA
    Ann N Y Acad Sci 996:101-11. 2003
  4. pmc Development of gene therapy for blood disorders: an update
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 122:1556-64. 2013
  5. pmc Pathophysiology and Clinical Manifestations of the β-Thalassemias
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Cold Spring Harb Perspect Med 2:a011726. 2012
  6. ncbi request reprint Assays to evaluate the genotoxicity of retroviral vectors
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38103, USA
    Mol Ther 14:459-60. 2006
  7. doi request reprint Development of gene therapy for blood disorders
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 111:4431-44. 2008
  8. 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
  9. doi request reprint Ninth Cooley's Anemia Symposium: summary and perspective
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Ann N Y Acad Sci 1202:248-54. 2010
  10. 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

Collaborators

Detail Information

Publications32

  1. ncbi request reprint Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood
    Hideki Hanawa
    Department of Hematology Oncology, Division of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Mol Ther 5:242-51. 2002
    ....
  2. ncbi request reprint Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia
    Hideki Hanawa
    332 North Lauderdale Dr, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 104:2281-90. 2004
    ..These data emphasize the importance of overcoming detrimental position effects for consistent therapeutic globin vector expression...
  3. ncbi request reprint Development of gene therapy for hemoglobin disorders
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, Tennessee 38105, USA
    Ann N Y Acad Sci 996:101-11. 2003
    ..These advances provide essential preclinical data which build toward the development of effective gene therapy for the severe hemoglobin disorders...
  4. pmc Development of gene therapy for blood disorders: an update
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 122:1556-64. 2013
    ..This approach should be successful in treating any disorder in which liver production of a specific protein is therapeutic. ..
  5. pmc Pathophysiology and Clinical Manifestations of the β-Thalassemias
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Cold Spring Harb Perspect Med 2:a011726. 2012
    ..Modern chelation regimens are effective in removing iron and preserving or restoring organ function...
  6. ncbi request reprint Assays to evaluate the genotoxicity of retroviral vectors
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38103, USA
    Mol Ther 14:459-60. 2006
  7. doi request reprint Development of gene therapy for blood disorders
    Arthur W Nienhuis
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 111:4431-44. 2008
    ..The hemoglobin disorders, an early target for gene therapy, have proved particularly challenging although ongoing research is yielding new information that may ultimately lead to successful clinical trials...
  8. 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...
  9. doi request reprint Ninth Cooley's Anemia Symposium: summary and perspective
    Arthur W Nienhuis
    Department of Hematology, St Jude Children s Research Hospital, Memphis, Tennessee, USA
    Ann N Y Acad Sci 1202:248-54. 2010
    ..Despite many advances, the global burden of disease for the thalassemias remains very high, with many challenges that still need to be addressed in order to optimize treatment for the majority of patients...
  10. 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...
  11. pmc An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation
    Byoung Y Ryu
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 111:1866-75. 2008
    ..Thus, this system is useful for comparing the safety profiles of vector cassettes with various regulatory elements for their potential for proto-oncogene activation...
  12. ncbi request reprint High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cells
    Hideki Hanawa
    Division of Experimental Hematology, St Jude Children s Research Hospital, 332 N Lauderdale, Memphis, TN 38105, USA
    Hum Gene Ther 13:2007-16. 2002
    ..Thus, these newly described vectors provide a means to achieve high-level gene expression, predominantly in erythroid cells, an outcome that may have potential therapeutic application...
  13. pmc Transduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteins
    Yoon Sang Kim
    Department of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee 38105, USA
    Mol Ther 18:1310-7. 2010
    ..These results support the use of the VSV-G envelope protein for the development of lentiviral producer cell lines for manufacture of clinical-grade vector...
  14. ncbi request reprint The degree of phenotypic correction of murine beta -thalassemia intermedia following lentiviral-mediated transfer of a human gamma-globin gene is influenced by chromosomal position effects and vector copy number
    Derek A Persons
    Division of Experimental Hematology, Department of Hematology and Oncology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 101:2175-83. 2003
    ..These data establish the potential of using a gamma-globin lentiviral vector for gene therapy of beta-thalassemia...
  15. ncbi request reprint Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells
    Derek A Persons
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, Memphis, TN, USA
    Blood 102:506-13. 2003
    ..These studies demonstrate that MGMT-based in vivo selection may be useful to increase genetically corrected cells to therapeutic levels in patients with beta-thalassemia...
  16. ncbi request reprint Defects in T-cell-mediated immunity to influenza virus in murine Wiskott-Aldrich syndrome are corrected by oncoretroviral vector-mediated gene transfer into repopulating hematopoietic cells
    Ted S Strom
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, 332 N Lauderdale, Memphis, TN 38105, USA
    Blood 102:3108-16. 2003
    ..The defective secondary T-cell response to influenza virus was also improved in gene-corrected animals...
  17. 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...
  18. pmc Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods
    Paolo Fagone
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Hum Gene Ther Methods 23:1-7. 2012
    ....
  19. ncbi request reprint WASP- mice exhibit defective immune responses to influenza A virus, Streptococcus pneumoniae, and Mycobacterium bovis BCG
    Samita Andreansky
    Department of Immunology, St Jude Children s Hospital, Memphis, TN 38104, USA
    Exp Hematol 33:443-51. 2005
    ..To quantify the immune response of WASP- mice to three different pathogens: influenza A virus, Streptococcus pneumoniae, and Mycobacterium bovis...
  20. ncbi request reprint Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector
    Marguerite V Evans-Galea
    Department of Experimental Hematology, St Jude Children s Research Hospital, Memphis, Tennessee 38105, USA
    Mol Ther 15:801-9. 2007
    ..This effect was diminished by including the insulator element in the vector genome...
  21. ncbi request reprint A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells
    Byoung Y Ryu
    Division of Experimental Hematology, Department of Hematology, St Jude Children s Research Hospital, 332 N Lauderdale St, Memphis, TN 38105, USA
    Blood Cells Mol Dis 39:221-8. 2007
    ..Promoter activation in K562 cells by vectors containing globin regulatory elements was significantly reduced by addition of flanking insulator elements...
  22. ncbi request reprint Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors
    Rachel M Koldej
    Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Hum Gene Ther Clin Dev 24:77-85. 2013
    ..We propose a clinical trial design in which cytokine-mobilized, autologous, transduced CD34(+) cells are administered after myelosuppression...
  23. ncbi request reprint Gene therapy for the hemoglobin disorders
    Derek A Persons
    St Jude Children s Research Hospital, 332 North Lauderdale Drive, Memphis, TN 38105, USA
    Curr Hematol Rep 2:348-55. 2003
    ..We review the developments in several areas that are critical for successful gene therapy of the hemoglobin disorders...
  24. pmc Mobilization and mechanism of transcription of integrated self-inactivating lentiviral vectors
    Hideki Hanawa
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, 332 N Lauderdale, Mail Stop 272, Memphis, Tennessee 38105, USA
    J Virol 79:8410-21. 2005
    ....
  25. ncbi request reprint In vivo selection to improve gene therapy of hematopoietic disorders
    Derek A Persons
    Department of Hematology and Oncology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Curr Opin Mol Ther 4:491-8. 2002
    ..Herein, we review recent progress in the development of in vivo selection systems, which hold promise in facilitating successful gene therapy...
  26. pmc Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial
    James A Allay
    Children s GMP, Memphis, TN 38105, USA
    Hum Gene Ther 22:595-604. 2011
    ..This material has been approved for use in clinical trials in the United States and the United Kingdom...
  27. ncbi request reprint Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells
    Thasia Leimig
    St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 99:3169-78. 2002
    ..The long-term expression and internalization of the PPCA by cells of systemic organs and the clear improvement of the neurologic phenotype support the use of this approach for the treatment of GS in humans. (Blood. 2002;99:3169-3178)..
  28. ncbi request reprint Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation
    Ted S Strom
    Division of Experimental Hematology, Department of Hematology Oncology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
    Blood 99:4626-8. 2002
    ..These results strengthen the analogy between murine and human WAS and provide a basis for the use of WAS-deficient mice to explore novel approaches for correction of the disease phenotype...
  29. 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...
  30. ncbi request reprint Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques
    Amit C Nathwani
    Department of Haematology, University College London, United Kingdom
    Blood 100:1662-9. 2002
    ..This nonhuman-primate model will be useful in further evaluation and development of rAAV vectors for gene therapy of hemophilia B...
  31. 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...
  32. ncbi request reprint Current status of gene therapy for hemophilia
    Amit C Nathwani
    Department of Haematology, University College London, 98 Chenies Mews, London WCIE 6HX, UK
    Curr Hematol Rep 2:319-27. 2003
    ..Recombinant adeno-associated viral vectors appear most promising for hemophilia gene therapy; however, this review summarizes all the major gene therapy approaches used and outlines the future challenges...