Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy
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Open Access
- 18 April 2002
- journal article
- clinical trial
- Published by Massachusetts Medical Society in New England Journal of Medicine
- Vol. 346 (16) , 1185-1193
- https://doi.org/10.1056/nejmoa012616
Abstract
X-linked severe combined immunodeficiency due to a mutation in the gene encoding the common γ (γc) chain is a lethal condition that can be cured by allogeneic stem-cell transplantation. We investigated whether infusion of autologous hematopoietic stem cells that had been transduced in vitro with the γc gene can restore the immune system in patients with severe combined immunodeficiency.Keywords
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