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Arthur W. Nienhuis, MD
Arthur W. Nienhuis, MD

Arthur W. Nienhuis, MD

Emeritus, St. Jude Faculty



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

Dr. Nienhuis served as Director and CEO of St. Jude Children's Research Hospital from 1993-2004.

Honors & Awards

  • American Society of Hematology Mentor Award (2009)
  • Henry Stratton Medal of the American Society of Hematology (1998)
  • National Cancer Advisory Board (Presidential appointment) (1998)
  • National Cancer Advisory Board (Presidential reappointment to a full six-year term) (1999)
  • Institute of Medicine of the National Academy of Sciences (now National Academy of Medicine) (2002)

Research Interests

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

Selected Publications

Nienhuis AW, Nathan DG. Pathophysiology and clinical manifestations of the β-Thalassemias. Cold Spring Harb Perspect Med 2(12):a011726, 2012.

Nienhuis AW, Persons DA. Development of gene therapy for thalassemia. Cold Spring Harb Perspect Med 2(11):doi:pii:a011833, 2012.

Hu H, Gomero E, Bonten E, Gray JT Allay J, Wu Y, Wu J, Calabrese C, Nienhuis A, d’Azzo A. Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis. Mol Ther 20(2):267-74, 2012.

Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O’Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 365(25):2357-65, 2011.

Allay JA, Sleep SE, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh J, Nienhuis AW, Davidoff AM, Nathwani A, Gray JT. Good manufacturing practice production of self-complementary serotype 8 AAV vector for a hemophilia B clinical trial. Hum Gene Ther 22(5):596-604. 2011.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Minogozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM. Long-term safety and efficacy following systemic administration of self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther 19(5):876-85. 2011.

Wilber A, Nienhuis AW, Persons DA. Transcriptional regulation of fetal to adult haemoglobin switching: new therapeutic opportunities. Blood 117(5):3945-53. 2011.

Nienhuis A. Ninth Cooley’s Anemia Symposium: Summary and Perspective. Annals of the New York Academy of Science 1202:248-54, 2010.

Wilber A, Hargrove, PW, Kim YS, Riberdy JM, Sankaran VG, Papanikolaou E, Georgomanoli M, Anagnou NP, Orkn SH, Nienhuis AW, Persons DA. Therapeutic levels of fetal hemoglobin in erythroid progeny of {beta}-thalassemic CD34+ cells following lentiviral vector-mediated gene transfer. Blood 117(10):2817-2826, 2010.

Kim Y-S, Wielgosz M, Hargrove P, Kepes S, Gray J, Persons D, Nienhuis A. Transduction of Human Primitive Repopulating Hematopoietic Cells with Lentiviral Vectors Pseudotyped with Various Envelope Proteins. Mol Ther 18(7):1310-7, 2010.

Wilber AC, Tschulena U, Hargrove PW, Kim Y-S, Persons DA, Barbas CF, Nienhuis AW. A zinc-finger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts. Blood 115(15):3033-3041, 2010.

Kim YJ, Kim YS, Larochelle A, Renaud G, Wolfsberg TG, Adler R, Donahue RE, Hermati P, Hong BK, Roayaei J, Akagi K, Riberdy JM, Nienhuis AW, Cunbar CE, Persons DA. 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(22):5434-43, 2009. PMID: 19339698.

Nienhuis AW. Development of gene therapy for blood disorders. Blood 111:4431-4444, 2008.

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(4):1866-1875, 2008. PMCID: PMC2234041

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.

Hanawa H, Persons DA, Nienhuis AW. Mobilization and mechanism of transcription of integrated self-inactivating (SIN) lentiviral vectors. J Virol July;79(13):8410-21, 2005.

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.

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

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 ß-thalassemia using in vivo selection of genetically-modified, drug resistant hematopoietic cells. Blood 102:506-513, 2003.

Kelly PF, Donahue RE, Vandergriff JA, Takatoku M, Bonifacino AC, Agricola BA, Metzger ME, Dunbar CE, Nienhuis AW, Vanin EF. Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vector. Blood Cells, Molecules & Diseases 30:132-143, 2003.

Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW. The degree of phenotypic correction of murine ß–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.

Last update: February 2013