title: [Correction of fatal genetic diseases using bone marrow transplantation. 2].
abstract: Genetic diseases can be treated by transplantation of normal allogeneic bone marrow. Four patients with congenital disorders of their hematopoietic and/or lymphoid stem cells have been transplanted. One patient had a congenital aplastic anemia (Blackfan-Diamond), one had Gaucher's disease, one had congenital agranulocytosis Kostmann and one had chronic granulomatous disease. All patients received busulfan, cyclophosphamide and total nodal irradiation as preparatory regimen. Total nodal irradiation and cyclophosphamide were used as immunosuppressive agents. In all cases hematopoietic ablation and donor lymphoid and hematopoietic engraftment were achieved. The basic disorders of all transplanted children could be corrected completely. All children are alive and well.
authors.affiliation: Abteilung für Hämatologie, Onkologie und Immunologie, Universitäts-Kinderklinik Jussuf Ibrahim, Jena.
authors.forename: F
authors.initials: F
authors.lastname: Zintl
authors.forename: J
authors.initials: J
authors.lastname: Hermann
authors.forename: D
authors.initials: D
authors.lastname: Fuchs
authors.forename: J
authors.initials: J
authors.lastname: Prager
authors.forename: A
authors.initials: A
authors.lastname: Müller
authors.forename: B
authors.initials: B
authors.lastname: Reiners
authors.forename: J
authors.initials: J
authors.lastname: Füller
mesh_terms: D000380:Agranulocytosis / Q000235:genetics* / Q000628:therapy; D000741:Anemia, Aplastic / Q000235:genetics* / Q000628:therapy; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D002675:Child, Preschool; D005776:Gaucher Disease / Q000235:genetics* / Q000628:therapy; D006085:Graft Survival / Q000187:drug effects; D006086:Graft vs Host Disease / Q000517:prevention & control; D006105:Granulomatous Disease, Chronic / Q000235:genetics* / Q000628:therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male
chemical_list: D007166:Immunosuppressive Agents
vernacular_title: Korrektur letal verlaufender genetischer Erkrankungen mit Hilfe der Knochenmarktransplantation. Teil 2.