Gene Therapy Method-National Institute of Allergy and Infectious Diseases (NIAID)-Chronic Granulomatous Disease

This ex vivo gene therapy approach, developed by the National Institute of Allergy and Infectious Diseases (NIAID), is designed to treat Chronic Granulomatous Disease (CGD), a rare primary immunodeficiency. The therapy involves the collection of autologous CD34+ hematopoietic stem cells, which are then transduced ex vivo with retroviral vectors—specifically MFGS-p47phox or MFGS-gp91phox—derived from the murine Moloney leukemia virus. These vectors deliver functional cDNA encoding either the p47phox protein (encoded by the NCF1 gene) for autosomal recessive CGD or the gp91phox protein (encoded by the CYBB gene) for X-linked CGD. The modified cells are reinfused into the patient, often following busulfan conditioning, to restore NADPH oxidase enzyme function in phagocytic cells. This restoration allows for the production of reactive oxygen species, which are essential for the immune system to kill bacterial and fungal pathogens, thereby preventing the recurrent life-threatening infections associated with CGD.

Other names

Gene Therapy Approach for Chronic Granulomatous Disease