Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

Inclusion Criteria: * males of all ages * severe WAS (clinical score 3-5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry * molecular confirmation by WAS gene DNA sequencing * lack of HLA-genotypically identical bone marrow or of a 10/10 antigen HLA-matched unrelated donor or cord blood after 3 month search * parental, guardian, patient signed informed consent/assent * willing to return for follow-up * only for patients who have received previous allogenic hematopoietic stem cell transplant: * failed allogenic hematopoietic stem cell transplant * contraindication to repeat transplantation Exclusion Criteria: * patient with HLA-genotypically identical bone marrow * patient with 10/10 antigen HLA-matched unrelated donor or cord blood * contraindication to leukapheresis * contraindication to bone marrow harvest * contraindication to administration of conditioning medication * HIV positive patient