X-linked chronic granulomatous disease (X-CGD) is an inherited immunodeficiency disease caused by a defect in the gp91phox gene encoding one of the subunits of the NADPH oxidase complex. NADPH oxidase plays an important role in eradicating the pathogen engulfed by the phagocytes. Therefore, CGD patients suffer from recurring life-threatening infection by bacteria or fungi, and die before 30 in most cases. In an effort to treat this life-threatening disease, we initiated a phase I/II gene therapy trial in 2007. Two X-CGD patients were enrolled in the trial. The retroviral vector used for gene delivery was the MLV-based MT vector containing gp91 phox cDNA (
Disclosures: Kim:ViroMed: Employment, Equity Ownership. Hong:ViroMed: Employment, Equity Ownership. Joo:ViroMed: Employment, Equity Ownership. Yoon:ViroMed: Employment, Equity Ownership. Kim:ViroMed: CEO, Equity Ownership.