[Correction of fatal genetic diseases using bone marrow transplantation. 1].
Bone marrow transplantation (BMT) was mainly used for the treatment of immunological, hematological and oncological diseases. Genetic diseases can also be treated by transplantation of normal histocompatible allogeneic bone marrow. This type of marrow transplantation is used for the treatment of inborn errors whose clinical expression is restricted to lymphoid or hematopoietic cells. The therapeutic role of bone marrow transplantation in the treatment of generalized genetic diseases is under investigation. Genetic disorders that can be cured include congenital immune deficiencies, infantile malignant osteopetrosis, thalassemia, infantile agranulocytosis, chronic granulomatous disease, lysosomal storage diseases and others. The use of autologous bone marrow after the insertion of a normal gene (gene therapy) in vitro circumvents the need for a histocompatible donor.
Zintl
F
F
Abteilung für Hämatologie, Onkologie und Immunologie, Universitäts-Kinderklinik Jussuf Ibrahim.
Hermann
J
J
Fuchs
D
D
Prager
J
J
Müller
A
A
Reiners
B
B
Füller
J
J
D016026:Bone Marrow Transplantation / Q000276:immunology / Q000379:methods*; D002648:Child; D015316:Genetic Therapy; D006402:Hematologic Diseases / Q000235:genetics* / Q000628:therapy; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics* / Q000628:therapy; D008661:Metabolism, Inborn Errors / Q000235:genetics* / Q000628:therapy
Korrektur letal verlaufender genetischer Erkrankung mit Hilfe der Knochenmarktransplantation. Teil 1.