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GTMB 7 - Gene Therapy & Molecular Biology

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<strong>Gene</strong> <strong>Therapy</strong> and <strong>Molecular</strong> <strong>Biology</strong> Vol 7, page 203Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F,Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova J,Bousso P, Le Deist F, Fischer A (2000) <strong>Gene</strong> therapy ofhuman severe combined immunodeficiency (SCID)-X1disease. Science 288, 669-672.Cavazzana-Calvo M, Hacein-Bey S, Yates F, De Villartay JP, LeDeist F, Fischer A (2001) <strong>Gene</strong> therapy of severe combinedimmunodeficiencies. J <strong>Gene</strong> Med 3, 201-206.Challita PM, Kohn DB (1994) Lack of expression from aretroviral vector after transduction of murine hematopoieticstem cells is associated with methylation in vivo. 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Microsc Res Tech 52,5-20.Emery AEH (1993) Duchenne muscular dystrophy. OxfordUniversity Press.Emery AEH (2002) The muscular dystrophies. Lancet 359, 687-695.Engelhardt JF, Schlossberg H, Yankaskas JR, Dudus L (1995)Progenitor cells of the adult human airway involved insubmucosal gland development. Development 121, 2031-2046.Engelhardt JF, Yankaskas JR, Ernst SA, Yang Y, Marino CR,Boucher RC, Cohn JA, Wilson JM (1992) Submucosalglands are the predominant site of CFTR expression in thehuman bronchus. 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Cambridge University Press,Cambridge, pp. 31-56.Friedmann T (2003) <strong>Gene</strong> therapy's new era: a balance ofunequivocal benefit and unequivocal harm. Mol Ther 8, 5-7.Furie B, Limentani SA, Rosenfield CG (1994) A practical guideto the evaluation and treatment of hemophilia. Blood 84, 3-9.Gaillard D, Ruocco S, Lallemand A, Dalemans W, Hinnrasky J,Puchelle E (1994) Immunohistochemical localization ofcystic fibrosis transmembrane conductance regulator inhuman fetal airway and digestive mucosa. Ped Res 36, 137-143.Galan HL, Bennett ML, Tyson RW, Owens G, Ragnault TR,Accurso F, Robbins JC, Schaack J (2002) Inefficienttransduction of sheep in utero after intra-amniotic injectionof retroviral producer cells. Am J Obstet Gynecol 187, 469-474.Gansbacher B, European Society of <strong>Gene</strong> <strong>Therapy</strong> (2003) Reportof a second serious adverse event in a clinical trial of genetherapy for X-linked severe combined immune deficiency(X-SCID). Position of the European Society of <strong>Gene</strong><strong>Therapy</strong> (ESGT). J <strong>Gene</strong> Med 5, 261-262.Geipel A, Berg C, Germer U, Krapp M, Kohl M, Gembruch U(2002) Mucopolysaccharidosis VII (Sly disease) as a causeof increased nuchal translucency and non-immune fetalhydrops: study of a family and technical approach to prenataldiagnosis in early and late pregnancy. Prenat Diagn 22, 493-495.203

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