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Gene Therapy for Hemophilia.

Nienhuis AW, Nathwani AC, Davidoff AM.

Mol Ther. 2017 May 3;25(5):1163-1167. doi: 10.1016/j.ymthe.2017.03.033. Epub 2017 Apr 11. Review.


Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector.

Singh S, Khan I, Khim S, Seymour B, Sommer K, Wielgosz M, Norgaard Z, Kiem HP, Adair J, Liggitt D, Nienhuis A, Rawlings DJ.

Mol Ther Methods Clin Dev. 2016 Dec 18;4:1-16. doi: 10.1016/j.omtm.2016.11.001. eCollection 2017 Mar 17.


Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia.

Zhao HF, Abraham A, Kim YS, Wang YD, Pestina T, Zhan J, Humphries K, Nienhuis AW, Persons DA.

Mol Ther. 2017 Mar 1;25(3):593-605. doi: 10.1016/j.ymthe.2017.01.019. Epub 2017 Feb 9.


The identification of hematopoietic-specific regulatory elements for WASp gene expression.

Zhan J, Johnson IM, Wielgosz M, Nienhuis AW.

Mol Ther Methods Clin Dev. 2016 Dec 14;3:16077. doi: 10.1038/mtm.2016.77. eCollection 2016. Review.


Gene Therapy for Hemophilia.

Nienhuis AW, Nathwani AC, Davidoff AM.

Hum Gene Ther. 2016 Apr;27(4):305-8. doi: 10.1089/hum.2016.018. Review.


Blood's 70th anniversary: 1988-1992.

Nienhuis AW.

Blood. 2016 Mar 3;127(9):1075-6. doi: 10.1182/blood-2015-10-638668. No abstract available.


Generation of a lentiviral vector producer cell clone for human Wiskott-Aldrich syndrome gene therapy.

Wielgosz MM, Kim YS, Carney GG, Zhan J, Reddivari M, Coop T, Heath RJ, Brown SA, Nienhuis AW.

Mol Ther Methods Clin Dev. 2015 Jan 21;2:14063. doi: 10.1038/mtm.2014.63. eCollection 2015.


Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.

N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.


Our journey to successful gene therapy for hemophilia B.

Nathwani AC, Nienhuis AW, Davidoff AM.

Hum Gene Ther. 2014 Nov;25(11):923-6. doi: 10.1089/hum.2014.2540. No abstract available.


Development of gene therapy for blood disorders: an update.

Nienhuis AW.

Blood. 2013 Aug 29;122(9):1556-64. doi: 10.1182/blood-2013-04-453209. Epub 2013 Jul 10. Review.


Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.

Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):77-85. doi: 10.1089/humc.2012.244. Epub 2013 Jun 20.


Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC.

Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.


Pathophysiology and Clinical Manifestations of the β-Thalassemias.

Nienhuis AW, Nathan DG.

Cold Spring Harb Perspect Med. 2012 Dec 1;2(12):a011726. doi: 10.1101/cshperspect.a011726.


Development of gene therapy for thalassemia.

Nienhuis AW, Persons DA.

Cold Spring Harb Perspect Med. 2012 Nov 1;2(11). pii: a011833. doi: 10.1101/cshperspect.a011833. Review.


Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods.

Fagone P, Wright JF, Nathwani AC, Nienhuis AW, Davidoff AM, Gray JT.

Hum Gene Ther Methods. 2012 Feb;23(1):1-7. doi: 10.1089/hgtb.2011.104.


Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

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.

N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.


Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.

Hu H, Gomero E, Bonten E, Gray JT, Allay J, Wu Y, Wu J, Calabrese C, Nienhuis A, d'Azzo A.

Mol Ther. 2012 Feb;20(2):267-74. doi: 10.1038/mt.2011.227. Epub 2011 Oct 18.


Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.

Allay JA, Sleep S, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh JH, Nienhuis AW, Davidoff AM, Nathwani AC, Gray JT.

Hum Gene Ther. 2011 May;22(5):595-604. doi: 10.1089/hum.2010.202.


Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities.

Wilber A, Nienhuis AW, Persons DA.

Blood. 2011 Apr 14;117(15):3945-53. doi: 10.1182/blood-2010-11-316893. Epub 2011 Feb 14. Review.


Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM.

Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18.


Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.

Wilber A, Hargrove PW, Kim YS, Riberdy JM, Sankaran VG, Papanikolaou E, Georgomanoli M, Anagnou NP, Orkin SH, Nienhuis AW, Persons DA.

Blood. 2011 Mar 10;117(10):2817-26. doi: 10.1182/blood-2010-08-300723. Epub 2010 Dec 14.


Ninth Cooley's Anemia Symposium: summary and perspective.

Nienhuis AW.

Ann N Y Acad Sci. 2010 Aug;1202:248-54. doi: 10.1111/j.1749-6632.2010.05549.x.


Transduction of human primitive repopulating hematopoietic cells with lentiviral vectors pseudotyped with various envelope proteins.

Kim YS, Wielgosz MM, Hargrove P, Kepes S, Gray J, Persons DA, Nienhuis AW.

Mol Ther. 2010 Jul;18(7):1310-7. doi: 10.1038/mt.2010.48. Epub 2010 Apr 6.


A zinc-finger transcriptional activator designed to interact with the gamma-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts.

Wilber A, Tschulena U, Hargrove PW, Kim YS, Persons DA, Barbas CF 3rd, Nienhuis AW.

Blood. 2010 Apr 15;115(15):3033-41. doi: 10.1182/blood-2009-08-240556. Epub 2010 Feb 26.


An integrated funding mechanism for clinical trials in human gene therapy.

Nienhuis A, Friedmann T.

Mol Ther. 2009 Jul;17(7):1117-8. doi: 10.1038/mt.2009.129. No abstract available.


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. doi: 10.1182/blood-2008-10-185199. Epub 2009 Apr 1.


The growing clinical impact of gene therapy.

Nienhuis A.

Mol Ther. 2008 Jun;16(6):995-6. doi: 10.1038/mt.2008.94. No abstract available.


Development of gene therapy for blood disorders.

Nienhuis AW.

Blood. 2008 May 1;111(9):4431-44. doi: 10.1182/blood-2007-11-078121. Review.


Lentiviral vectors for the treatment of Wiskott-Aldrich syndrome.

Nienhuis AW.

Gene Ther. 2005 Apr;12(7):555-6. No abstract available.


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.


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.


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.


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.


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.


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.


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.


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.


Current status and prospects for gene therapy.

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

Vox Sang. 2004 Aug;87(2):73-81. Review.


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.


Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia.

Kiem HP, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Adler R, Kuramoto K, Calmels B, Bonifacino A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Nienhuis A, Blau CA, Andrews RG, Donahue RE, Dunbar CE.

Mol Ther. 2004 Mar;9(3):389-95.


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.


Gene therapy for the hemoglobin disorders.

Persons DA, Nienhuis AW.

Curr Hematol Rep. 2003 Jul;2(4):348-55. Review.


Current status of gene therapy for hemophilia.

Nathwani AC, Nienhuis AW, Davidoff AM.

Curr Hematol Rep. 2003 Jul;2(4):319-27. Review.


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.


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.


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.


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.


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