In utero transplantation for genetical diseases
F. Porta*, F. Bolda, E. Baggio, S. Berta, R. Baffelli
M. Ridella, S. Guarisco, V. Grassi, D. de Martis, K. Tettoni
L.D. Notarangelo, A. Lanfranchi
Centro di Terapia Cellulare e Genica delle Oncoemopatie Infantili "A.Nocivelli", Spedali Civili, Brescia, Italy *
Bone marrow transplantation (BMT) is the therapy of choice for several genetic diseases of blood. Since 1968 there is evidence that stem cell transplantation can cure diseases characterized by lack or miss-function of blood cells. Infact the first correction of a genetic disease by stem cells infusion has been realized in a child affected by a form of immunodeficiency , severe combined immunodeficiency (SCID), in a child who lacked T cells.
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