Gene therapy for chronic granulomatous disease

Chronic granulomatous disease (CGD) is a rare inherited immunodeficiency characterized by recurrent, often life threatening bacterial and fungal infections due to a functional defect in the microbial-killing activity of phagocytic neutrophils. If regular care and conventional therapy fail, the disease can be cured by bone marrow transplantation. This treatment is, however, only available to patients with human leukocyte antigen-identical sibling or matched unrelated donors. One therapeutic option for patients lacking suitable donors is the genetic modification of autologous hematopoietic stem cells. This review discusses the developments that have led to the realization of a successful gene therapy protocol for the correction of CGD.