PubMed

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector-transduced CD34+ cells.

Kim YJ, Kim YS, Larochelle A, Renaud G, Wolfsberg TG, Adler R, Donahue RE, Hematti P, Hong BK, Roayaei J, Akagi K, Riberdy JM, Nienhuis AW, Dunbar CE, Persons DA.

Blood. 2009 May 28;113(22):5434-43. Epub 2009 Apr 1.PMID: 19339698 [PubMed - indexed for MEDLINE]Related articles

Development of gene therapy for blood disorders.

Nienhuis AW.

Blood. 2008 May 1;111(9):4431-44. Review.PMID: 18441245 [PubMed - indexed for MEDLINE]Related articlesFree article

Lentiviral vectors for the treatment of Wiskott-Aldrich syndrome.

Nienhuis AW.

Gene Ther. 2005 Apr;12(7):555-6. No abstract available. PMID: 18360950 [PubMed - indexed for MEDLINE]Related articles

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation.

Ryu BY, Evans-Galea MV, Gray JT, Bodine DM, Persons DA, Nienhuis AW.

Blood. 2008 Feb 15;111(4):1866-75. Epub 2007 Nov 8.PMID: 17991809 [PubMed - indexed for MEDLINE]Related articlesFree article

A chromatin insulator blocks interactions between globin regulatory elements and cellular promoters in erythroid cells.

Ryu BY, Persons DA, Evans-Galea MV, Gray JT, Nienhuis AW.

Blood Cells Mol Dis. 2007 Nov-Dec;39(3):221-8. Epub 2007 Jun 29.PMID: 17601756 [PubMed - indexed for MEDLINE]Related articles

Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector.

Evans-Galea MV, Wielgosz MM, Hanawa H, Srivastava DK, Nienhuis AW.

Mol Ther. 2007 Apr;15(4):801-9. Epub 2007 Feb 13.PMID: 17299406 [PubMed - indexed for MEDLINE]Related articles

Assays to evaluate the genotoxicity of retroviral vectors.

Nienhuis AW.

Mol Ther. 2006 Oct;14(4):459-60. Epub 2006 Aug 21. No abstract available. PMID: 16920033 [PubMed - indexed for MEDLINE]Related articles

Genotoxicity of retroviral integration in hematopoietic cells.

Nienhuis AW, Dunbar CE, Sorrentino BP.

Mol Ther. 2006 Jun;13(6):1031-49. Epub 2006 Apr 19. Review.PMID: 16624621 [PubMed - indexed for MEDLINE]Related articles

Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque.

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.

Blood. 2006 May 15;107(10):3865-7. Epub 2006 Jan 26.PMID: 16439674 [PubMed - indexed for MEDLINE]Related articlesFree article

Mobilization and mechanism of transcription of integrated self-inactivating lentiviral vectors.

Hanawa H, Persons DA, Nienhuis AW.

J Virol. 2005 Jul;79(13):8410-21.PMID: 15956585 [PubMed - indexed for MEDLINE]Related articlesFree article

WASP- mice exhibit defective immune responses to influenza A virus, Streptococcus pneumoniae, and Mycobacterium bovis BCG.

Andreansky S, Liu H, Turner S, McCullers JA, Lang R, Rutschman R, Doherty PC, Murray PJ, Nienhuis AW, Strom TS.

Exp Hematol. 2005 Apr;33(4):443-51.PMID: 15781335 [PubMed - indexed for MEDLINE]Related articles

Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells.

Hematti P, Hong BK, 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.

PLoS Biol. 2004 Dec;2(12):e423. Epub 2004 Nov 23.PMID: 15550989 [PubMed - indexed for MEDLINE]Related articlesFree article

Current status and prospects for gene therapy.

Nathwani AC, Benjamin R, Nienhuis AW, Davidoff AM.

Vox Sang. 2004 Aug;87(2):73-81. Review.PMID: 15355497 [PubMed - indexed for MEDLINE]Related articles

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia.

Hanawa H, Hargrove PW, Kepes S, Srivastava DK, Nienhuis AW, Persons DA.

Blood. 2004 Oct 15;104(8):2281-90. Epub 2004 Jun 15.PMID: 15198957 [PubMed - indexed for MEDLINE]Related articlesFree article

Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system.

Hanawa H, Hematti P, Keyvanfar K, Metzger ME, Krouse A, Donahue RE, Kepes S, Gray J, Dunbar CE, Persons DA, Nienhuis AW.

Blood. 2004 Jun 1;103(11):4062-9. Epub 2004 Feb 19.PMID: 14976042 [PubMed - indexed for MEDLINE]Related articlesFree article

Gene therapy for the hemoglobin disorders.

Persons DA, Nienhuis AW.

Curr Hematol Rep. 2003 Jul;2(4):348-55. Review.PMID: 12901333 [PubMed - indexed for MEDLINE]Related articles

Current status of gene therapy for hemophilia.

Nathwani AC, Nienhuis AW, Davidoff AM.

Curr Hematol Rep. 2003 Jul;2(4):319-27. Review.PMID: 12901329 [PubMed - indexed for MEDLINE]Related articles

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.

Strom TS, Turner SJ, Andreansky S, Liu H, Doherty PC, Srivastava DK, Cunningham JM, Nienhuis AW.

Blood. 2003 Nov 1;102(9):3108-16. Epub 2003 Jul 10.PMID: 12855574 [PubMed - indexed for MEDLINE]Related articlesFree article

Development of gene therapy for hemoglobin disorders.

Nienhuis AW, Hanawa H, Sawai N, Sorrentino BP, Persons DA.

Ann N Y Acad Sci. 2003 May;996:101-11.PMID: 12799288 [PubMed - indexed for MEDLINE]Related articles

Functional correction of T cells derived from patients with the Wiskott-Aldrich syndrome (WAS) by transduction with an oncoretroviral vector encoding the WAS protein.

Strom TS, Gabbard W, Kelly PF, Cunningham JM, Nienhuis AW.

Gene Ther. 2003 May;10(9):803-9.PMID: 12704420 [PubMed - indexed for MEDLINE]Related articles

Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34 positive cells marked with a RD114 pseudotyped oncoretroviral vector.

Kelly PF, Donahue RE, Vandergriff JA, Takatoku M, Bonifacino AC, Agricola BA, Metzger ME, Dunbar CE, Nienhuis AW, Vanin EF.

Blood Cells Mol Dis. 2003 Jan-Feb;30(1):132-43.PMID: 12667996 [PubMed - indexed for MEDLINE]Related articles

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.

Persons DA, Allay ER, Sawai N, Hargrove PW, Brent TP, Hanawa H, Nienhuis AW, Sorrentino BP.

Blood. 2003 Jul 15;102(2):506-13. Epub 2003 Mar 27.PMID: 12663444 [PubMed - indexed for MEDLINE]Related articlesFree article

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.

Hanawa H, Persons DA, Nienhuis AW.

Hum Gene Ther. 2002 Nov 20;13(17):2007-16.PMID: 12489996 [PubMed - indexed for MEDLINE]Related articles

Novel therapeutic approaches in sickle cell disease.

Walters MC, Nienhuis AW, Vichinsky E.

Hematology Am Soc Hematol Educ Program. 2002:10-34. Review.PMID: 12446417 [PubMed - indexed for MEDLINE]Related articlesFree article

In vivo selection to improve gene therapy of hematopoietic disorders.

Persons DA, Nienhuis AW.

Curr Opin Mol Ther. 2002 Oct;4(5):491-8.PMID: 12435051 [PubMed - indexed for MEDLINE]Related articles

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.

Persons DA, Hargrove PW, Allay ER, Hanawa H, Nienhuis AW.

Blood. 2003 Mar 15;101(6):2175-83. Epub 2002 Oct 31.PMID: 12411297 [PubMed - indexed for MEDLINE]Related articlesFree article

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.

Nathwani AC, Davidoff AM, Hanawa H, Hu Y, Hoffer FA, Nikanorov A, Slaughter C, Ng CY, Zhou J, Lozier JN, Mandrell TD, Vanin EF, Nienhuis AW.

Blood. 2002 Sep 1;100(5):1662-9.PMID: 12176886 [PubMed - indexed for MEDLINE]Related articlesFree article

Correction of the murine Wiskott-Aldrich syndrome phenotype by hematopoietic stem cell transplantation.

Strom TS, Li X, Cunningham JM, Nienhuis AW.

Blood. 2002 Jun 15;99(12):4626-8.PMID: 12036897 [PubMed - indexed for MEDLINE]Related articlesFree article

Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells.

Leimig T, Mann L, Martin Mdel P, Bonten E, Persons D, Knowles J, Allay JA, Cunningham J, Nienhuis AW, Smeyne R, d'Azzo A.

Blood. 2002 May 1;99(9):3169-78.PMID: 11964280 [PubMed - indexed for MEDLINE]Related articlesFree article

Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood.

Hanawa H, Kelly PF, Nathwani AC, Persons DA, Vandergriff JA, Hargrove P, Vanin EF, Nienhuis AW.

Mol Ther. 2002 Mar;5(3):242-51.PMID: 11863413 [PubMed - indexed for MEDLINE]Related articlesFree article

Gene Therapy 2000.

Williams DA, Nienhuis AW, Hawley RG, Smith FO.

Hematology Am Soc Hematol Educ Program. 2000:376-393.PMID: 11701552 [PubMed - as supplied by publisher]Related articlesFree article

Bone marrow-derived cells contribute to tumor neovasculature and, when modified to express an angiogenesis inhibitor, can restrict tumor growth in mice.

Davidoff AM, Ng CY, Brown P, Leary MA, Spurbeck WW, Zhou J, Horwitz E, Vanin EF, Nienhuis AW.

Clin Cancer Res. 2001 Sep;7(9):2870-9.PMID: 11555605 [PubMed - indexed for MEDLINE]Related articlesFree article

Autocrine expression of both endostatin and green fluorescent protein provides a synergistic antitumor effect in a murine neuroblastoma model.

Davidoff AM, Leary MA, Ng CY, Spurbeck WW, Frare P, Vanhove M, Nienhuis AW, Vanin EF.

Cancer Gene Ther. 2001 Jul;8(7):537-45.PMID: 11498776 [PubMed - indexed for MEDLINE]Related articlesFree article

Functional requirements for phenotypic correction of murine beta-thalassemia: implications for human gene therapy.

Persons DA, Allay ER, Sabatino DE, Kelly P, Bodine DM, Nienhuis AW.

Blood. 2001 May 15;97(10):3275-82.PMID: 11342459 [PubMed - indexed for MEDLINE]Related articlesFree article

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

Nathwani AC, Davidoff A, Hanawa H, Zhou JF, Vanin EF, Nienhuis AW.

Blood. 2001 Mar 1;97(5):1258-65.PMID: 11222368 [PubMed - indexed for MEDLINE]Related articlesFree article

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.

Kelly PF, Vandergriff J, Nathwani A, Nienhuis AW, Vanin EF.

Blood. 2000 Aug 15;96(4):1206-14.PMID: 10942359 [PubMed - indexed for MEDLINE]Related articlesFree article

Gene therapy for the hemoglobin disorders: past, present, and future.

Persons DA, Nienhuis AW.

Proc Natl Acad Sci U S A. 2000 May 9;97(10):5022-4. Review. No abstract available. PMID: 10805762 [PubMed - indexed for MEDLINE]Related articlesFree article

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.

Nathwani AC, Hanawa H, Vandergriff J, Kelly P, Vanin EF, Nienhuis AW.

Gene Ther. 2000 Feb;7(3):183-95.PMID: 10694794 [PubMed - indexed for MEDLINE]Related articlesFree article

Retrovirus mediated gene transfer of the self antigen MBP into the bone marrow of mice alters resistance to experimental autoimmune encephalomyelitis.

Peters TR, Bodine DM, McDonagh KT, Lovett-Racke A, McFarland HF, McFarlin DE, Nienhuis AW, Racke MK.

J Neuroimmunol. 2000 Feb 1;103(1):51-62.PMID: 10674989 [PubMed - indexed for MEDLINE]Related articles

High levels of lymphoid expression of enhanced green fluorescent protein in nonhuman primates transplanted with cytokine-mobilized peripheral blood CD34(+) cells.

Donahue RE, Wersto RP, Allay JA, Agricola BA, Metzger ME, Nienhuis AW, Persons DA, Sorrentino BP.

Blood. 2000 Jan 15;95(2):445-52.PMID: 10627448 [PubMed - indexed for MEDLINE]Related articlesFree article

An olfactory receptor gene is located in the extended human beta-globin gene cluster and is expressed in erythroid cells.

Feingold EA, Penny LA, Nienhuis AW, Forget BG.

Genomics. 1999 Oct 1;61(1):15-23.PMID: 10512676 [PubMed - indexed for MEDLINE]Related articles

Adenovirus-mediated expresssion of the murine ecotropic receptor facilitates transduction of human hematopoietic cells with an ecotropic retroviral vector.

Nathwani AC, Persons DA, Stevenson SC, Frare P, McClelland A, Nienhuis AW, Vanin EF.

Gene Ther. 1999 Aug;6(8):1456-68.PMID: 10467370 [PubMed - indexed for MEDLINE]Related articlesFree article

Enforced expression of the GATA-2 transcription factor blocks normal hematopoiesis.

Persons DA, Allay JA, Allay ER, Ashmun RA, Orlic D, Jane SM, Cunningham JM, Nienhuis AW.

Blood. 1999 Jan 15;93(2):488-99.PMID: 9885210 [PubMed - indexed for MEDLINE]Related articlesFree article

Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells.

Persons DA, Allay JA, Riberdy JM, Wersto RP, Donahue RE, Sorrentino BP, Nienhuis AW.

Nat Med. 1998 Oct;4(10):1201-5. No abstract available. PMID: 9771757 [PubMed - indexed for MEDLINE]Related articles

Targeted integration of a recombinant globin gene adeno-associated viral vector into human chromosome 19.

Bertran J, Yang Y, Hargrove P, Vanin EF, Nienhuis AW.

Ann N Y Acad Sci. 1998 Jun 30;850:163-77.PMID: 9668538 [PubMed - indexed for MEDLINE]Related articles

An improved method for generating retroviral producer clones for vectors lacking a selectable marker gene.

Persons DA, Mehaffey MG, Kaleko M, Nienhuis AW, Vanin EF.

Blood Cells Mol Dis. 1998 Jun;24(2):167-82.PMID: 9642098 [PubMed - indexed for MEDLINE]Related articles

Retroviral-mediated transfer of the green fluorescent protein gene into murine hematopoietic cells facilitates scoring and selection of transduced progenitors in vitro and identification of genetically modified cells in vivo.

Persons DA, Allay JA, Allay ER, Smeyne RJ, Ashmun RA, Sorrentino BP, Nienhuis AW.

Blood. 1997 Sep 1;90(5):1777-86.PMID: 9292510 [PubMed - indexed for MEDLINE]Related articlesFree article

High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3' gamma globin gene regulatory element and integrates as tandem copies in erythroid cells.

Hargrove PW, Vanin EF, Kurtzman GJ, Nienhuis AW.

Blood. 1997 Mar 15;89(6):2167-75.PMID: 9058741 [PubMed - indexed for MEDLINE]Related articlesFree article

Gene transfer into hematopoietic cells.

Nienhuis AW, Bertran J, Hargrove P, Vanin E, Yang Y.

Stem Cells. 1997;15 Suppl 1:123-34. Review.PMID: 9368332 [PubMed - indexed for MEDLINE]Related articlesFree article

Recombinant adeno-associated virus-mediated high-efficiency, transient expression of the murine cationic amino acid transporter (ecotropic retroviral receptor) permits stable transduction of human HeLa cells by ecotropic retroviral vectors.

Bertran J, Miller JL, Yang Y, Fenimore-Justman A, Rueda F, Vanin EF, Nienhuis AW.

J Virol. 1996 Oct;70(10):6759-66.PMID: 8794313 [PubMed - indexed for MEDLINE]Related articlesFree article