Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)
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The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.
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Ages Eligible for Study:
1 Year to 18 Years (Child, Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
x-linked Chronic Granulomatous Disease
history of life-threatening severe infections
no HLA-matched related or unrelated donor
therapy resistent life threatening infections/organ dysfunction
no other treatment options e.g. HSCT
> 18 years of age
life expectancy > 2 years
infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)