Gene therapy for chronic granulomatous disease

A Kume, MC Dinauer - Journal of Laboratory and Clinical Medicine, 2000 - Elsevier
Recent progress in the development of gene therapy for chronic granulomatous disease
(CGD), an inherited immunodeficiency syndrome, is reviewed. This disorder results from
defects in any of the four genes encoding essential subunits of respiratory burst oxidase, the
superoxide-generating enzyme complex in phagocytic leukocytes. The absence of
respiratory burst oxidants results in recurrent bacterial and fungal infections and can also be
complicated by the formation of inflammatory granulomas. Although current management …