Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy

Gene therapy was used in five boys with X-linked severe combined immunodeficiency disease. In this disorder, a mutation disables the common γ (γc) chain, a component of five cytokine receptors that are essential for the development of T cells and natural killer cells. The disease is fatal within the...

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Veröffentlicht in:	The New England journal of medicine 2002-04, Vol.346 (16), p.1185-1193
Hauptverfasser:	Hacein-Bey-Abina, Salima, Le Deist, Françoise, Carlier, Frédérique, Bouneaud, Cécile, Hue, Christophe, De Villartay, Jean-Pierre, Thrasher, Adrian J, Wulffraat, Nicolas, Sorensen, Ricardo, Dupuis-Girod, Sophie, Fischer, Alain, Davies, E. Graham, Kuis, Wietse, Leiva, Lilly, Cavazzana-Calvo, Marina
Format:	Artikel
Sprache:	eng
Schlagworte:	Antibodies - blood Antigens, CD - analysis Biological and medical sciences Bone marrow Bone Marrow Cells gc gene Genetic Linkage Genetic Therapy Genetic Vectors Humans Immune system Immunodeficiencies Immunodeficiencies. Immunoglobulinopathies Immunoglobulins - blood Immunoglobulins, Intravenous Immunopathology Infant Killer Cells, Natural - physiology Lymphocyte Count Male Medical sciences Moloney murine leukemia virus - genetics Receptors, Cytokine - genetics Severe Combined Immunodeficiency - genetics Severe Combined Immunodeficiency - immunology Severe Combined Immunodeficiency - therapy Stem cells Streptococcus infections T-Lymphocytes - immunology T-Lymphocytes - physiology Transduction, Genetic Transgenes X Chromosome X-linked severe combined immunodeficiency
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