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Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy.

Adair JE, Waters T, Haworth KG, Kubek SP, Trobridge GD, Hocum JD, Heimfeld S, Kiem HP.

Nat Commun. 2016 Oct 20;7:13173. doi: 10.1038/ncomms13173.


Advances of gene therapy for primary immunodeficiencies.

Candotti F.

F1000Res. 2016 Mar 9;5. pii: F1000 Faculty Rev-310. doi: 10.12688/f1000research.7512.1. eCollection 2016. Review.


Optimizing autologous cell grafts to improve stem cell gene therapy.

Psatha N, Karponi G, Yannaki E.

Exp Hematol. 2016 Jul;44(7):528-39. doi: 10.1016/j.exphem.2016.04.007. Epub 2016 Apr 19. Review.


Engraftment and lineage potential of adult hematopoietic stem and progenitor cells is compromised following short-term culture in the presence of an aryl hydrocarbon receptor antagonist.

Gu A, Torres-Coronado M, Tran CA, Vu H, Epps EW, Chung J, Gonzalez N, Blanchard S, DiGiusto DL.

Hum Gene Ther Methods. 2014 Aug;25(4):221-31. doi: 10.1089/hgtb.2014.043.


Evaluation of engraftment and immunological tolerance after reduced intensity conditioning in a rhesus hematopoietic stem cell gene therapy model.

Uchida N, Weitzel RP, Evans ME, Green R, Bonifacino AC, Krouse AE, Metzger ME, Hsieh MM, Donahue RE, Tisdale JF.

Gene Ther. 2014 Feb;21(2):148-57. doi: 10.1038/gt.2013.67. Epub 2013 Nov 21.


Foamy virus vectors for HIV gene therapy.

Olszko ME, Trobridge GD.

Viruses. 2013 Oct 22;5(10):2585-600. doi: 10.3390/v5102585. Review.


Hematopoietic stem cell gene therapy:assessing the relevance of preclinical models.

Larochelle A, Dunbar CE.

Semin Hematol. 2013 Apr;50(2):101-30. Review. No abstract available.


Large animal models for foamy virus vector gene therapy.

Trobridge GD, Horn PA, Beard BC, Kiem HP.

Viruses. 2012 Dec 7;4(12):3572-88. doi: 10.3390/v4123572. Review.


Characterization of the effects of exercise training on hematopoietic stem cell quantity and function.

De Lisio M, Parise G.

J Appl Physiol (1985). 2012 Nov;113(10):1576-84. doi: 10.1152/japplphysiol.00717.2012. Epub 2012 Sep 27.


Stem cell factor-displaying simian immunodeficiency viral vectors together with a low conditioning regimen allow for long-term engraftment of gene-marked autologous hematopoietic stem cells in macaques.

Verhoeyen E, Relouzat F, Cambot M, Costa C, Nègre D, Legrand F, Joubert C, Le Grand R, Cosset FL, Leboulch P, Dubart-Kupperschmitt A, Prost S.

Hum Gene Ther. 2012 Jul;23(7):754-68. doi: 10.1089/hum.2012.020. Epub 2012 Jul 11.


Hematopoietic stem cell expansion and gene therapy.

Watts KL, Adair J, Kiem HP.

Cytotherapy. 2011 Nov;13(10):1164-71. doi: 10.3109/14653249.2011.620748. Review.


Mesenchymal stem cells in ex vivo cord blood expansion.

Robinson SN, Simmons PJ, Yang H, Alousi AM, Marcos de Lima J, Shpall EJ.

Best Pract Res Clin Haematol. 2011 Mar;24(1):83-92. doi: 10.1016/j.beha.2010.11.001. Epub 2011 Feb 23. Review.


Contributions of gene marking to cell and gene therapies.

Barese CN, Dunbar CE.

Hum Gene Ther. 2011 Jun;22(6):659-68. doi: 10.1089/hum.2010.237. Epub 2011 May 5. Review.


Gene therapy of chronic granulomatous disease: the engraftment dilemma.

Grez M, Reichenbach J, Schwäble J, Seger R, Dinauer MC, Thrasher AJ.

Mol Ther. 2011 Jan;19(1):28-35. doi: 10.1038/mt.2010.232. Epub 2010 Nov 2. Review.


Ex vivo expansion of retrovirally transduced primate CD34+ cells results in overrepresentation of clones with MDS1/EVI1 insertion sites in the myeloid lineage after transplantation.

Sellers S, Gomes TJ, Larochelle A, Lopez R, Adler R, Krouse A, Donahue RE, Childs RW, Dunbar CE.

Mol Ther. 2010 Sep;18(9):1633-9. doi: 10.1038/mt.2010.117. Epub 2010 Jun 22.


Large animal models of hematopoietic stem cell gene therapy.

Trobridge GD, Kiem HP.

Gene Ther. 2010 Aug;17(8):939-48. doi: 10.1038/gt.2010.47. Epub 2010 Apr 29. Review.


Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils.

Kang EM, Choi U, Theobald N, Linton G, Long Priel DA, Kuhns D, Malech HL.

Blood. 2010 Jan 28;115(4):783-91. doi: 10.1182/blood-2009-05-222760. Epub 2009 Dec 1.


Development of a human immunodeficiency virus type 1-based lentiviral vector that allows efficient transduction of both human and rhesus blood cells.

Uchida N, Washington KN, Hayakawa J, Hsieh MM, Bonifacino AC, Krouse AE, Metzger ME, Donahue RE, Tisdale JF.

J Virol. 2009 Oct;83(19):9854-62. doi: 10.1128/JVI.00357-09. Epub 2009 Jul 22.


Noninvasive bioluminescent imaging demonstrates long-term multilineage engraftment of ex vivo-expanded CD34-selected umbilical cord blood cells.

Steiner D, Gelovani J, Savoldo B, Robinson SN, Decker WK, Brouard N, Najjar A, Xing D, Yang H, Li S, Marini F, Zweidler-McKay PA, Bollard CM, Shpall EJ, Dotti G, Simmons PJ.

Stem Cells. 2009 Aug;27(8):1932-40. doi: 10.1002/stem.111.


Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primate.

Hayakawa J, Ueda T, Lisowski L, Hsieh MM, Washington K, Phang O, Metzger M, Krouse A, Donahue RE, Sadelain M, Tisdale JF.

Hum Gene Ther. 2009 Jun;20(6):563-72. doi: 10.1089/hum.2008.186.

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