Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children

Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children

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.

Inclusion Criteria:

• 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

Exclusion Criteria:

• > 18 years of age
• HIV infection
• life expectancy > 2 years
• infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)