Gene Therapy for Chronic Granulomatous Disease
Gene Therapy for Chronic Granulomatous Disease
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)patients 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 non-related donor

 -  therapy resistent life threatening infections/organ dysfunction

 -  no other treatment options e.g. BMT

 Exclusion Criteria:

 -  < 18 years of age

 -  HIV infection

 -  life expectancy > 2 years

 -  infections treatable by conventional therapy (antibiotics, allogeneic granulocytes)