Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

@article{Gaspar2004GeneTO,
  title={Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector},
  author={H Bobby Gaspar and Kathryn L. Parsley and Steven J Howe and Douglas King and Kimberly C. Gilmour and Jo Sinclair and Gaby S. Brouns and Manfred Schmidt and Christof von Kalle and Torben Barington and Marianne A Jakobsen and Hans Ole Christensen and Abdulaziz Al Ghonaium and Harry N White and John L. Smith and Roland J. Levinsky and Robin R Ali and Christine Kinnon and Adrian James Thrasher},
  journal={The Lancet},
  year={2004},
  volume={364},
  pages={2181-2187}
}
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 associated with substantial morbidity and mortality. We investigated the application of… Expand
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