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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.


Data in support of transcriptional regulation and function of Fas-antisense long noncoding RNA during human erythropoiesis.

Villamizar O, Chambers CB, Mo YY, Torry DS, Hofstrand R, Riberdy JM, Persons DA, Wilber A.

Data Brief. 2016 Apr 7;7:1288-95. doi: 10.1016/j.dib.2016.03.106. eCollection 2016 Jun.


Fas-antisense long noncoding RNA is differentially expressed during maturation of human erythrocytes and confers resistance to Fas-mediated cell death.

Villamizar O, Chambers CB, Mo YY, Torry DS, Hofstrand R, Riberdy JM, Persons DA, Wilber A.

Blood Cells Mol Dis. 2016 May;58:57-66. doi: 10.1016/j.bcmd.2016.03.002. Epub 2016 Mar 3.


Long noncoding RNA Saf and splicing factor 45 increase soluble Fas and resistance to apoptosis.

Villamizar O, Chambers CB, Riberdy JM, Persons DA, Wilber A.

Oncotarget. 2016 Mar 22;7(12):13810-26. doi: 10.18632/oncotarget.7329.


Increased Engraftment of Human Short Term Repopulating Hematopoietic Cells in NOD/SCID/IL2rγnull Mice by Lentiviral Expression of NUP98-HOXA10HD.

Abraham A, Kim YS, Zhao H, Humphries K, Persons DA.

PLoS One. 2016 Jan 13;11(1):e0147059. doi: 10.1371/journal.pone.0147059. eCollection 2016.


Amelioration of murine sickle cell disease by nonablative conditioning and γ-globin gene-corrected bone marrow cells.

Pestina TI, Hargrove PW, Zhao H, Mead PE, Smeltzer MP, Weiss MJ, Wilber A, Persons DA.

Mol Ther Methods Clin Dev. 2015 Dec 2;2:15045. doi: 10.1038/mtm.2015.45. eCollection 2015.


Low-level GATA2 overexpression promotes myeloid progenitor self-renewal and blocks lymphoid differentiation in mice.

Nandakumar SK, Johnson K, Throm SL, Pestina TI, Neale G, Persons DA.

Exp Hematol. 2015 Jul;43(7):565-77.e1-10. doi: 10.1016/j.exphem.2015.04.002. Epub 2015 Apr 20.


Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells.

Urbinati F, Hargrove PW, Geiger S, Romero Z, Wherley J, Kaufman ML, Hollis RP, Chambers CB, Persons DA, Kohn DB, Wilber A.

Exp Hematol. 2015 May;43(5):346-351. doi: 10.1016/j.exphem.2015.01.009. Epub 2015 Feb 12.


High-titer foamy virus vector transduction and integration sites of human CD34(+) cell-derived SCID-repopulating cells.

Nasimuzzaman M, Kim YS, Wang YD, Persons DA.

Mol Ther Methods Clin Dev. 2014 Jun 4;1:14020. doi: 10.1038/mtm.2014.20. eCollection 2014.


Nfix is a novel regulator of murine hematopoietic stem and progenitor cell survival.

Holmfeldt P, Pardieck J, Saulsberry AC, Nandakumar SK, Finkelstein D, Gray JT, Persons DA, McKinney-Freeman S.

Blood. 2013 Oct 24;122(17):2987-96. doi: 10.1182/blood-2013-04-493973. Epub 2013 Sep 16.


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.


High-efficiency transduction of rhesus hematopoietic repopulating cells by a modified HIV1-based lentiviral vector.

Uchida N, Hargrove PW, Lap CJ, Evans ME, Phang O, Bonifacino AC, Krouse AE, Metzger ME, Nguyen AD, Hsieh MM, Wolfsberg TG, Donahue RE, Persons DA, Tisdale JF.

Mol Ther. 2012 Oct;20(10):1882-92. doi: 10.1038/mt.2012.159. Epub 2012 Aug 7.


Cell Membrane-associated heparan sulfate is a receptor for prototype foamy virus in human, monkey, and rodent cells.

Nasimuzzaman M, Persons DA.

Mol Ther. 2012 Jun;20(6):1158-66. doi: 10.1038/mt.2012.41. Epub 2012 Mar 20.


Hydroxyurea therapy of a murine model of sickle cell anemia inhibits the progression of pneumococcal disease by down-modulating E-selectin.

Lebensburger JD, Howard T, Hu Y, Pestina TI, Gao G, Johnson M, Zakharenko SS, Ware RE, Tuomanen EI, Persons DA, Rosch JW.

Blood. 2012 Feb 23;119(8):1915-21. doi: 10.1182/blood-2011-08-374447. Epub 2011 Nov 30.


Therapeutic in vivo selection of thymic-derived natural T regulatory cells following non-myeloablative hematopoietic stem cell transplant for IPEX.

Kasow KA, Morales-Tirado VM, Wichlan D, Shurtleff SA, Abraham A, Persons DA, Riberdy JM.

Clin Immunol. 2011 Nov;141(2):169-76. doi: 10.1016/j.clim.2011.07.005. Epub 2011 Aug 2.


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.


Solving the problem of γ-retroviral vectors containing long terminal repeats.

Persons DA, Baum C.

Mol Ther. 2011 Feb;19(2):229-31. doi: 10.1038/mt.2010.305. No abstract available.


Gene therapy for canine leukocyte adhesion deficiency with lentiviral vectors using the murine stem cell virus and human phosphoglycerate kinase promoters.

Hunter MJ, Zhao H, Tuschong LM, Bauer TR Jr, Burkholder TH, Persons DA, Hickstein DD.

Hum Gene Ther. 2011 Jun;22(6):689-96. doi: 10.1089/hum.2010.130. Epub 2011 Apr 11.


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.


Gene therapy: Targeting β-thalassaemia.

Persons DA.

Nature. 2010 Sep 16;467(7313):277-8. doi: 10.1038/467277a. No abstract available.


Analysis of alpha hemoglobin stabilizing protein overexpression in murine β-thalassemia.

Nasimuzzaman M, Khandros E, Wang X, Kong Y, Zhao H, Weiss D, Rivella S, Weiss MJ, Persons DA.

Am J Hematol. 2010 Oct;85(10):820-2. doi: 10.1002/ajh.21829. Erratum in: Am J Hematol. 2011 Sep;86(9):824.


The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in beta-thalassemia patients.

Persons DA.

Ann N Y Acad Sci. 2010 Aug;1202:69-74. doi: 10.1111/j.1749-6632.2010.05581.x. Review.


Lentiviral vector gene therapy: effective and safe?

Persons DA.

Mol Ther. 2010 May;18(5):861-2. doi: 10.1038/mt.2010.70. No abstract available.


Hydroxyurea therapy requires HbF induction for clinical benefit in a sickle cell mouse model.

Lebensburger JD, Pestina TI, Ware RE, Boyd KL, Persons DA.

Haematologica. 2010 Sep;95(9):1599-603. doi: 10.3324/haematol.2010.023325. Epub 2010 Apr 7.


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.


Statins protect against fulminant pneumococcal infection and cytolysin toxicity in a mouse model of sickle cell disease.

Rosch JW, Boyd AR, Hinojosa E, Pestina T, Hu Y, Persons DA, Orihuela CJ, Tuomanen EI.

J Clin Invest. 2010 Feb;120(2):627-35. doi: 10.1172/JCI39843. Epub 2010 Jan 19.


Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.

Persons DA.

Hematology Am Soc Hematol Educ Program. 2009:690-7. doi: 10.1182/asheducation-2009.1.690. Review.


Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene.

Zhao H, Pestina TI, Nasimuzzaman M, Mehta P, Hargrove PW, Persons DA.

Blood. 2009 Jun 4;113(23):5747-56. doi: 10.1182/blood-2008-10-186684. Epub 2009 Apr 13.


Genetically modified CD34+ hematopoietic stem cells contribute to turnover of brain perivascular macrophages in long-term repopulated primates.

Soulas C, Donahue RE, Dunbar CE, Persons DA, Alvarez X, Williams KC.

Am J Pathol. 2009 May;174(5):1808-17. doi: 10.2353/ajpath.2009.081010. Epub 2009 Apr 6.


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.


Optimized lentiviral vector design improves titer and transgene expression of vectors containing the chicken beta-globin locus HS4 insulator element.

Hanawa H, Yamamoto M, Zhao H, Shimada T, Persons DA.

Mol Ther. 2009 Apr;17(4):667-74. doi: 10.1038/mt.2009.1. Epub 2009 Feb 17.


Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin.

Pestina TI, Hargrove PW, Jay D, Gray JT, Boyd KM, Persons DA.

Mol Ther. 2009 Feb;17(2):245-52. doi: 10.1038/mt.2008.259. Epub 2008 Dec 2.


Progress toward safe and effective gene therapy for beta-thalassemia and sickle cell disease.

Lebensburger J, Persons DA.

Curr Opin Drug Discov Devel. 2008 Mar;11(2):225-32. Review.


Globin lentiviral vector insertions can perturb the expression of endogenous genes in beta-thalassemic hematopoietic cells.

Hargrove PW, Kepes S, Hanawa H, Obenauer JC, Pei D, Cheng C, Gray JT, Neale G, Persons DA.

Mol Ther. 2008 Mar;16(3):525-33. doi: 10.1038/ Epub 2008 Jan 15.


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.


Role of erythropoietin receptor signaling in Friend virus-induced erythroblastosis and polycythemia.

Zhang J, Randall MS, Loyd MR, Li W, Schweers RL, Persons DA, Rehg JE, Noguchi CT, Ihle JN, Ney PA.

Blood. 2006 Jan 1;107(1):73-8. Epub 2005 Sep 20.


Transgenic mice with pancellular enhanced green fluorescent protein expression in primitive hematopoietic cells and all blood cell progeny.

Dominici M, Tadjali M, Kepes S, Allay ER, Boyd K, Ney PA, Horwitz E, Persons DA.

Genesis. 2005 May;42(1):17-22.


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.


Gene therapy for the hemoglobin disorders.

Persons DA, Tisdale JF.

Semin Hematol. 2004 Oct;41(4):279-86. Review.


Hematopoietic cells and osteoblasts are derived from a common marrow progenitor after bone marrow transplantation.

Dominici M, Pritchard C, Garlits JE, Hofmann TJ, Persons DA, Horwitz EM.

Proc Natl Acad Sci U S A. 2004 Aug 10;101(32):11761-6. Epub 2004 Jul 28.


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.


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.


Update on gene therapy for hemoglobin disorders.

Persons DA.

Curr Opin Mol Ther. 2003 Oct;5(5):508-16. Review.


Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors.

Persons DA, Allay JA, Bonifacino A, Lu T, Agricola B, Metzger ME, Donahue RE, Dunbar CE, Sorrentino BP.

Blood. 2004 Feb 1;103(3):796-803. Epub 2003 Aug 14.


Gene therapy for the hemoglobin disorders.

Persons DA, Nienhuis AW.

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


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.


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