Gene Therapy for X-linked Chronic Granulomatous Disease

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.