Cure of Children with X-
SCID
l Most successful human trial to date
l T cells lack C cytokine receptor required for lymphoid
proliferation
l Retroviral transfer of C cytokine receptor gene into CD34+
cells
l Autotransplantation
l Selection of corrected cells
l Normal immune function in 7/9 patients
l T cell leukemia (clonal) in 2/9 patients 3 years post-
transduction