Arthur Nienhuis, MD

Arthur W. Nienhuis, MD

Member, St. Jude Faculty

Departments

Hematology

Divisions

Experimental Hematology


Education

MD - School of Medicine, University of California at Los Angeles


Research Interests

  • Genetic therapy of hematological diseases
  • Regulation of hematopoiesis and hemoglobin synthesis

Selected Publications

Nienhuis AW. Development of gene therapy for blood disorders. Blood 2008 (Epub ahead of print)

Ryu BY, Evans-Galea MV, Gray JT, Persons DA, Bodine DM, Nienhuis AW. An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation. Blood 111:1866-1875, 2008.

Ryu BY, Evans-Galea MV, Gray JT, Persons DA, Bodine DM, Nienhuis AW. A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells. Blood Cells, Mol Dis 39:221-8, 2007.

Evans-Galea MV, Wielgosz MM, Hanawa H, Srivastava DK, Nienhuis AW. Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector. Mol Ther 15:801-9, 2007.

Seggewiss R, Pittaluga S, Adler RL, Guenaga FJ, Ferguson C, Pilz IH, Ryu B, Sorrentino BP, Young WS 3rd, Donahue RE, von Kalle C, Nienhuis AW, Dunbar CE. Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Blood 107:3865-7, 2006.

Andeansky S, Liu H, Turner S, McCullers JA, Lang R, Rutschman R, Doherty PC, Murray PJ, Nienhuis AW, Strom TS. WASP-mice exhibit defective immune responses to influenza A virus, Streptococcus pneumoniae, and Mycobacterium bovis BCG. Exp Hematol 33:443-51, 2005.

Hanawa H, Persons DA, Nienhuis AW. Mobilization and mechanism of transcription of integrated self-inactivating (SIN) lentiviral vectors. J Virol 79:8410-21, 2005.

Hanawa H, Hargrove PW, Kepes S, Srivastava DK, Nienhuis AW, Persons DA. Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia. Blood 104:2281-2290, 2004.

Hematti P, Hong B-K, Ferguson C, Adler R, Hanawa H, Sellers S, Holt IE, Eckfeldt CE, Sharma Y, Schmidt M, von Kalle C, Persons DA, Billings EM, Verfaillie CM, Nienhuis AW, Wolfsberg TG, Dunbar CE, Calmels B.  Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells.  PLoS Biol Dec;2(12), 2004.

Hanawa H, Hematti P, Keyvanfar K, Metzger ME, Krouse A, Donahue RE, Kepes S, Gray J, Dunbar CE, Persons DA, Nienhuis AW. Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system. Blood 103:4062-4069, 2004.

Kiem H-P, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Adler R, Kuramoto K, Calmels B, Bonifacion A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Nienhuis AW, Blau A, Andrews RG, Donahue RE, Dunbar C.  Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells:  no progression to clonal hematopoiesis or leukemia. Mol Ther 9:389-95, 2004.

Nathwani AC, Nienhuis AW, Davidoff AM. Current status of gene therapy for hemophilia. Curr Hematol Rep 2:319-327, 2003.

Persons DA, Nienhuis AW. Gene therapy for the hemoglobin disorders. Curr Hematol Rep 2:348-355, 2003.

Nienhuis AW, Hanawa H, Sawai N, Sorrentino BP, Persons DA. Development of gene therapy for hemoglobin disorders. Ann N Y Acad Sci 996:101-111, 2003.

Strom TS, Turner SJ, Andreansky S, Liu H, Doherty PC, Srivastava DK, Cunningham JM, Nienhuis AW. 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. Blood 102:3108-3116, 2003.

Strom TS, Gabbard W, Kelly PF, Cunningham JM, Nienhuis AW. Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein. Gene Ther 10:803-809, 2003.

Persons DA, Allay ER, Sawai N, Hargrove PW, Brent TP, Hanawa H, Nienhuis AW, Sorrentino BP. Successful treatment of murine β-thalassemia using in vivo selection of genetically-modified, drug resistant hematopoietic cells. Blood 102:506-513, 2003.

Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW. The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number. Blood 101:2175-2183, 2003.

Hanawa H, Persons DA, Nienhuis AW. High level erythroid lineage-directed gene expression using globin gene regulatory elements following lentiviral vector mediated gene transfer into primitive human and murine hematopoietic cells. Hum Gene Ther 13: 2007-2016, 2002.

Nathwani AC, Davidoff AM, Hanawa H, Hu Y, Hoffer FA, Nikanorov A, Slaughter C, Ng CYC, Zhou J, Lozier JA, Mandrell TD, Vanin EF, Nienhuis AW. 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. Blood 100:1662-1669, 2002.

Strom TS, Li X, Cunningham JM, Nienhuis AW. Correction of the murine Wiskott-Aldrich Syndrome (WAS) phenotype by hematopoietic stem cell transplantation. Blood 99:4626-4628, 2002.

Hanawa H, Kelly PF, Nathwani AC, Persons DA, Carrington J, Hargrove P, Vanin EF, Nienhuis AW. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol Therapy 5:242-251, 2002.

Persons DA, Allay ER, Sabatino DE, Kelly P, Bodine DM, Nienhuis AW.  Functional requirements for phenotypic correction of murine b-thalassemia:  implications for human gene therapy. Blood 97:3275-3282, 2001.

Nathwani A, Davidoff A, Hanawa H, Zhou J-F, Vanin EF, Nienhuis AW. Factors influencing in-vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. Blood 97:1258-1265, 2001.

Nathwani AC, Hanawa H, Vandergriff J, Kelly P, Vanin EF, Nienhuis AW. Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild type AAV. Gene Ther 7:183-195, 2000.

Kelly PF, Vandergriff J, Nathwani A, Nienhuis AW, Vanin EF. Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. Blood 96:1206-1214, 2000.

Persons DA, Allay JA, Allay ER, Ashmun RA, Cunningham JM, Nienhuis AW. Enforced expression of the GATA-2 transcription factor blocks normal hematopoiesis. Blood 93:488-499, 1999.

Persons DA, Allay JA, Riberdy JM, Wersto RP, Donahue RE, Sorrentino BP, Nienhuis AW. Use of the green fluorescent protein gene as a marker to identify and track genetically modified hematopoietic cells. Nature Medicine 4:1201-1205, 1998.

Last update: March 2008

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