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459 related items for PubMed ID: 8399494

  • 1. Transfer of the ADA gene into bone marrow cells and peripheral blood lymphocytes for the treatment of patients affected by ADA-deficient SCID.
    Bordignon C, Mavilio F, Ferrari G, Servida P, Ugazio AG, Notarangelo LD, Gilboa E, Rossini S, O'Reilly RJ, Smith CA.
    Hum Gene Ther; 1993 Aug; 4(4):513-20. PubMed ID: 8399494
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  • 2. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments].
    Smogorzewska EM, Weinberg KI, Kohn DB.
    Med Wieku Rozwoj; 2003 Aug; 7(1):27-34. PubMed ID: 13130167
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  • 3. Treatment of severe combined immunodeficiency disease (SCID) due to adenosine deaminase deficiency with CD34+ selected autologous peripheral blood cells transduced with a human ADA gene. Amendment to clinical research project, Project 90-C-195, January 10, 1992.
    Blaese RM, Culver KW, Chang L, Anderson WF, Mullen C, Nienhuis A, Carter C, Dunbar C, Leitman S, Berger M.
    Hum Gene Ther; 1993 Aug; 4(4):521-7. PubMed ID: 7691188
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  • 9. Retrovirus mediated gene transfer as therapy for adenosine deaminase (ADA) deficiency.
    Ramsey WJ, Mullen CA, Blaese RM.
    Leukemia; 1995 Oct; 9 Suppl 1():S70. PubMed ID: 7475319
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  • 10. Bone marrow transplantation and alternatives for adenosine deaminase deficiency.
    Gaspar HB.
    Immunol Allergy Clin North Am; 2010 May; 30(2):221-36. PubMed ID: 20493398
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  • 11. PEG-ADA: an alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency.
    Hershfield MS.
    Hum Mutat; 1995 May; 5(2):107-12. PubMed ID: 7749407
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  • 12. Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice.
    Carriglio N, Klapwijk J, Hernandez RJ, Vezzoli M, Chanut F, Lowe R, Draghici E, Nord M, Albertini P, Cristofori P, Richards J, Staton H, Appleby J, Aiuti A, Sauer AV.
    Hum Gene Ther Clin Dev; 2017 Mar; 28(1):17-27. PubMed ID: 28319446
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  • 14. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients.
    Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P, Mazzolari E, Maggioni D, Rossi C, Servida P, Ugazio AG, Mavilio F.
    Science; 1995 Oct 20; 270(5235):470-5. PubMed ID: 7570000
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  • 15. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.
    Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ.
    Mol Ther; 2006 Oct 20; 14(4):505-13. PubMed ID: 16905365
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  • 16. Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.
    Otsu M, Yamada M, Nakajima S, Kida M, Maeyama Y, Hatano N, Toita N, Takezaki S, Okura Y, Kobayashi R, Matsumoto Y, Tatsuzawa O, Tsuchida F, Kato S, Kitagawa M, Mineno J, Hershfield MS, Bali P, Candotti F, Onodera M, Kawamura N, Sakiyama Y, Ariga T.
    J Clin Immunol; 2015 May 20; 35(4):384-98. PubMed ID: 25875699
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  • 17. Bone marrow gene transfer in three patients with adenosine deaminase deficiency.
    Hoogerbrugge PM, van Beusechem VW, Fischer A, Debree M, le Deist F, Perignon JL, Morgan G, Gaspar B, Fairbanks LD, Skeoch CH, Moseley A, Harvey M, Levinsky RJ, Valerio D.
    Gene Ther; 1996 Feb 20; 3(2):179-83. PubMed ID: 8867866
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  • 20. Treatment of patients with severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency by autologous transplantation of genetically modified bone marrow cells.
    Hoogerbrugge PM, Vossen JM, v Beusechem VW, Valerio D.
    Hum Gene Ther; 1992 Oct 20; 3(5):553-8. PubMed ID: 1420454
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