To raise awareness of StemCellAwarenessDay, here is a phase 1/2 trial investigating long-term safety and efficacy of lentiviral hematopietic stemcell gene therapy for Wiskottaldrichsyndrome
) was to assess the efficacy of HSC gene therapy in patients with WAS, based on clinical improvement in at least one of the following parameters : eczema status, the frequency and severity of infections, bruising and bleeding episodes, and autoimmune disorders and the number of disease-related days of hospitalization.
The exclusion criteria were HLA-genotypically identical bone marrow; 10/10 or 9/10 antigen HLA-matched unrelated donor or HLA-matched cord blood; contraindication to leukapheresis; contraindication to bone marrow harvest; contraindication to conditioning medication; and HIV positivity.
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