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Items: 1 to 20 of 202

1.

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.

2.

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.

3.

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.

5.

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.

PMID:
28190779
6.

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.

7.

A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.

Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D, Malik P.

Blood. 2009 Aug 6;114(6):1174-85. doi: 10.1182/blood-2009-01-201863. Epub 2009 May 27.

8.

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.

9.

The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.

Papanikolaou E, Georgomanoli M, Stamateris E, Panetsos F, Karagiorga M, Tsaftaridis P, Graphakos S, Anagnou NP.

Hum Gene Ther. 2012 Jan;23(1):15-31. doi: 10.1089/hum.2011.048. Epub 2011 Dec 5.

10.

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.

Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S.

PLoS One. 2012;7(3):e32345. doi: 10.1371/journal.pone.0032345. Epub 2012 Mar 27.

11.

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.

12.

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.

Traxler EA, Yao Y, Wang YD, Woodard KJ, Kurita R, Nakamura Y, Hughes JR, Hardison RC, Blobel GA, Li C, Weiss MJ.

Nat Med. 2016 Sep;22(9):987-90. doi: 10.1038/nm.4170. Epub 2016 Aug 15.

13.

Pharmacological Induction of Human Fetal Globin Gene in Hydroxyurea-Resistant Primary Adult Erythroid Cells.

Chou YC, Chen RL, Lai ZS, Song JS, Chao YS, Shen CK.

Mol Cell Biol. 2015 Jul;35(14):2541-53. doi: 10.1128/MCB.00035-15. Epub 2015 May 18.

14.

Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing.

Xu J, Peng C, Sankaran VG, Shao Z, Esrick EB, Chong BG, Ippolito GC, Fujiwara Y, Ebert BL, Tucker PW, Orkin SH.

Science. 2011 Nov 18;334(6058):993-6. doi: 10.1126/science.1211053. Epub 2011 Oct 13.

15.

Monomethylfumarate induces γ-globin expression and fetal hemoglobin production in cultured human retinal pigment epithelial (RPE) and erythroid cells, and in intact retina.

Promsote W, Makala L, Li B, Smith SB, Singh N, Ganapathy V, Pace BS, Martin PM.

Invest Ophthalmol Vis Sci. 2014 May 13;55(8):5382-93. doi: 10.1167/iovs.14-14179.

16.
17.

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.

18.

Forced TR2/TR4 expression in sickle cell disease mice confers enhanced fetal hemoglobin synthesis and alleviated disease phenotypes.

Campbell AD, Cui S, Shi L, Urbonya R, Mathias A, Bradley K, Bonsu KO, Douglas RR, Halford B, Schmidt L, Harro D, Giacherio D, Tanimoto K, Tanabe O, Engel JD.

Proc Natl Acad Sci U S A. 2011 Nov 15;108(46):18808-13. doi: 10.1073/pnas.1104964108. Epub 2011 Oct 31.

19.

Correction of sickle cell disease by homologous recombination in embryonic stem cells.

Wu LC, Sun CW, Ryan TM, Pawlik KM, Ren J, Townes TM.

Blood. 2006 Aug 15;108(4):1183-8. Epub 2006 Apr 25.

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