Research Topics
Species | Arthur W NienhuisSummaryAffiliation: St. Jude Children's Research Hospital Country: USA Publications
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Detail Information
Publications
Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human bloodHideki Hanawa
Department of Hematology/Oncology, Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA
Mol Ther 5:242-51. 2002....- Development of gene therapy for hemoglobin disordersArthur 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... - Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemiaHideki 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... 
Pathophysiology and Clinical Manifestations of the β-ThalassemiasArthur 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...
Development of gene therapy for blood disordersArthur 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...- Ninth Cooley's Anemia Symposium: summary and perspectiveArthur 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... - Genotoxicity of retroviral integration in hematopoietic cellsArthur 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... - Assays to evaluate the genotoxicity of retroviral vectorsArthur 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 - Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vectorPatrick 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... - An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activationByoung 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... - High-level erythroid lineage-directed gene expression using globin gene regulatory elements after lentiviral vector-mediated gene transfer into primitive human and murine hematopoietic cellsHideki 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... - Transduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteinsYoon 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... - 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 numberDerek 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... - Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cellsDerek 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... - 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 cellsTed 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... - Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector systemHideki 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... - Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methodsPaolo Fagone
Department of Hematology, St Jude Children s Research Hospital, Memphis, TN 38105, USA
Hum Gene Ther Methods 23:1-7. 2012.... - WASP- mice exhibit defective immune responses to influenza A virus, Streptococcus pneumoniae, and Mycobacterium bovis BCGSamita 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... - Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vectorMarguerite 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... - A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cellsByoung 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... - Gene therapy for the hemoglobin disordersDerek 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... - Mobilization and mechanism of transcription of integrated self-inactivating lentiviral vectorsHideki 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.... - In vivo selection to improve gene therapy of hematopoietic disordersDerek 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... - Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trialJames 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... - Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantationTed 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... - Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cellsThasia 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).. - Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cellsPeiman 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... - Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaqueRuth 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... - Current status of gene therapy for hemophiliaAmit 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... - Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaquesAmit 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...