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Page 1
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.
Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, Muul L, Morgan RA, Anderson WF. Blaese RM, et al. Science. 1995 Oct 20;270(5235):475-80. doi: 10.1126/science.270.5235.475. Science. 1995. PMID: 7570001 Clinical Trial.
Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.
Muul LM, Tuschong LM, Soenen SL, Jagadeesh GJ, Ramsey WJ, Long Z, Carter CS, Garabedian EK, Alleyne M, Brown M, Bernstein W, Schurman SH, Fleisher TA, Leitman SF, Dunbar CE, Blaese RM, Candotti F. Muul LM, et al. Blood. 2003 Apr 1;101(7):2563-9. doi: 10.1182/blood-2002-09-2800. Epub 2002 Nov 27. Blood. 2003. PMID: 12456496 Free article. Clinical Trial.
T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.
Kohn DB, Hershfield MS, Carbonaro D, Shigeoka A, Brooks J, Smogorzewska EM, Barsky LW, Chan R, Burotto F, Annett G, Nolta JA, Crooks G, Kapoor N, Elder M, Wara D, Bowen T, Madsen E, Snyder FF, Bastian J, Muul L, Blaese RM, Weinberg K, Parkman R. Kohn DB, et al. Nat Med. 1998 Jul;4(7):775-80. doi: 10.1038/nm0798-775. Nat Med. 1998. PMID: 9662367 Free PMC article.
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
Candotti F, Shaw KL, Muul L, Carbonaro D, Sokolic R, Choi C, Schurman SH, Garabedian E, Kesserwan C, Jagadeesh GJ, Fu PY, Gschweng E, Cooper A, Tisdale JF, Weinberg KI, Crooks GM, Kapoor N, Shah A, Abdel-Azim H, Yu XJ, Smogorzewska M, Wayne AS, Rosenblatt HM, Davis CM, Hanson C, Rishi RG, Wang X, Gjertson D, Yang OO, Balamurugan A, Bauer G, Ireland JA, Engel BC, Podsakoff GM, Hershfield MS, Blaese RM, Parkman R, Kohn DB. Candotti F, et al. Blood. 2012 Nov 1;120(18):3635-46. doi: 10.1182/blood-2012-02-400937. Epub 2012 Sep 11. Blood. 2012. PMID: 22968453 Free PMC article. Clinical Trial.
Preferential survival of CD4+ T lymphocytes engineered with anti-human immunodeficiency virus (HIV) genes in HIV-infected individuals.
Morgan RA, Walker R, Carter CS, Natarajan V, Tavel JA, Bechtel C, Herpin B, Muul L, Zheng Z, Jagannatha S, Bunnell BA, Fellowes V, Metcalf JA, Stevens R, Baseler M, Leitman SF, Read EJ, Blaese RM, Lane HC. Morgan RA, et al. Hum Gene Ther. 2005 Sep;16(9):1065-74. doi: 10.1089/hum.2005.16.1065. Hum Gene Ther. 2005. PMID: 16149905
53 results