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Journal Abstract Search

210 related items for PubMed ID: 21851067

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  • 2. Improved lentiviral vectors for Wiskott-Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis.
    Frecha C, Toscano MG, Costa C, Saez-Lara MJ, Cosset FL, Verhoeyen E, Martin F.
    Gene Ther; 2008 Jun; 15(12):930-41. PubMed ID: 18323794
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  • 3. A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.
    Charrier S, Stockholm D, Seye K, Opolon P, Taveau M, Gross DA, Bucher-Laurent S, Delenda C, Vainchenker W, Danos O, Galy A.
    Gene Ther; 2005 Apr; 12(7):597-606. PubMed ID: 15616597
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  • 9. Expression of human Wiskott-Aldrich syndrome protein in patients' cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins.
    Huang MM, Tsuboi S, Wong A, Yu XJ, Oh-Eda M, Derry JM, Francke U, Fukuda M, Weinberg KI, Kohn DB.
    Gene Ther; 2000 Feb; 7(4):314-20. PubMed ID: 10694812
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  • 10. Optimization of adenovirus serotype 35 vectors for efficient transduction in human hematopoietic progenitors: comparison of promoter activities.
    Sakurai F, Kawabata K, Yamaguchi T, Hayakawa T, Mizuguchi H.
    Gene Ther; 2005 Oct; 12(19):1424-33. PubMed ID: 15944730
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  • 11. Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS.
    Zanta-Boussif MA, Charrier S, Brice-Ouzet A, Martin S, Opolon P, Thrasher AJ, Hope TJ, Galy A.
    Gene Ther; 2009 May; 16(5):605-19. PubMed ID: 19262615
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  • 12. Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.
    Braun CJ, Boztug K, Paruzynski A, Witzel M, Schwarzer A, Rothe M, Modlich U, Beier R, Göhring G, Steinemann D, Fronza R, Ball CR, Haemmerle R, Naundorf S, Kühlcke K, Rose M, Fraser C, Mathias L, Ferrari R, Abboud MR, Al-Herz W, Kondratenko I, Maródi L, Glimm H, Schlegelberger B, Schambach A, Albert MH, Schmidt M, von Kalle C, Klein C.
    Sci Transl Med; 2014 Mar 12; 6(227):227ra33. PubMed ID: 24622513
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  • 13. Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences.
    Martín F, Toscano MG, Blundell M, Frecha C, Srivastava GK, Santamaría M, Thrasher AJ, Molina IJ.
    Gene Ther; 2005 Apr 12; 12(8):715-23. PubMed ID: 15750617
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  • 15. Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.
    Charrier S, Dupré L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O, Cattaneo F, Aiuti A, Eckenberg R, Thrasher AJ, Roncarolo MG, Galy A.
    Gene Ther; 2007 Mar 12; 14(5):415-28. PubMed ID: 17051251
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  • 16. Gene therapy of primary T cell immunodeficiencies.
    Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M.
    Gene; 2013 Aug 10; 525(2):170-3. PubMed ID: 23583799
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  • 20. Efficient transcriptional targeting of human hematopoietic stem cells and blood cell lineages by lentiviral vectors containing the regulatory element of the Wiskott-Aldrich syndrome gene.
    Leuci V, Gammaitoni L, Capellero S, Sangiolo D, Mesuraca M, Bond HM, Migliardi G, Cammarata C, Aglietta M, Morrone G, Piacibello W.
    Stem Cells; 2009 Nov 10; 27(11):2815-23. PubMed ID: 19785032
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