Article
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector.
Lancet (London, England) - 1 Jan 2000
Gaspar H Bobby, Parsley Kathryn L, Howe Steven, King Doug, Gilmour Kimberly C, Sinclair Joanna, Brouns Gaby, Schmidt Manfred, Von Kalle Christof, Barington Torben, Jakobsen Marianne A, Christensen Hans O, Al Ghonaium Abdulaziz, White Harry N, Smith John L, Levinsky Roland J, Ali Robin R, Kinnon Christine, Thrasher Adrian J
Abstract excerpt
BACKGROUND: X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the common cytokine-receptor gamma chain (gamma(c)), resulting in disruption of development of T lymphocytes and natural-killer cells. B-lymphocyte function is also intrinsically compromised. Allogeneic bone-marrow transplantation is successful if HLA-matched family donors are available, but HLA-mismatched procedures are...
Topics
- Antigens, CD34
- Bone Marrow Cells
- Bone Marrow Transplantation
- Child, Preschool
- Gammaretrovirus
- Gene Transfer Techniques
- Genetic Diseases, X-Linked
- Genetic Therapy