Clinical Research Directory

Inclusion Criteria: * Male X-CGD patients \>23 months of age. Youngest patients (\>1 month and ≤ 23 months) may be enrolled at physician's appreciation; in this case mobilization of peripheral HSC may be replaced by two bone marrow harvests. * Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction \> 70% of the biochemical activity of the NAHPD-oxidase. * At least one ongoing or resistant or at high risk of relapse severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy. * No HLA-matched donor available after 3 months search, unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable. * No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBs Ag positive) or hepatitis C virus (anti-HCV Ab positive). * Written informed consent for adult patient. * Parental/guardian and where appropriate child's signed consent/assent. Exclusion Criteria: * 10/10 HLA identical (A, B, C, DR, DQ) family or unrelated. * Contraindication for leukapheresis (anaemia Hb \<8g/dl, cardiovascular instability, severe coagulopathy). * Contraindication for administration of conditioning medication and any component of the Investigational Medicinal Product (IMP) preparation. * Administration of gamma interferon within 30 days before the infusion of transduced autologous CD34+ cells. * Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period. * Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study. * Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements.