Bone marrow gene transfer in three patients with adenosine deaminase deficiency

Autor: Hoogerbrugge, P M, van Beusechem, V W, Fischer, A, Debree, M, le Deist, F, Perignon, J L, Morgan, G, Gaspar, B, Fairbanks, L D, Skeoch, C H, Moseley, A, Harvey, M, Levinsky, R J, Valerio, D

Publikováno v: Hoogerbrugge, P M, van Beusechem, V W, Fischer, A, Debree, M, le Deist, F, Perignon, J L, Morgan, G, Gaspar, B, Fairbanks, L D, Skeoch, C H, Moseley, A, Harvey, M, Levinsky, R J & Valerio, D 1996, ' Bone marrow gene transfer in three patients with adenosine deaminase deficiency ', Gene Therapy, vol. 3, no. 2, pp. 179-83 .
Gene Therapy, 3(2), 179-83. Nature Publishing Group

Adenosine deaminase (ADA) deficiency results in severe combined immune deficiency disease (SCID), which is fatal without treatment. Allogeneic bone marrow transplantation (BMT) is the treatment of choice if an HLA-identical sibling bone marrow donor

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Bone marrow gene transfer in three patients with adenosine deaminase deficiency.

Autor: Hoogerbrugge PM; Department of Medical Biochemistry, University of Leiden, Rijswijk, Netherlands., van Beusechem VW, Fischer A, Debree M, le Deist F, Perignon JL, Morgan G, Gaspar B, Fairbanks LD, Skeoch CH, Moseley A, Harvey M, Levinsky RJ, Valerio D

Publikováno v: Gene therapy [Gene Ther] 1996 Feb; Vol. 3 (2), pp. 179-83.