Abstract
X-linked chronic granulomatous disease (X-CGD) is a rare, devastating primary immunodeficiency disorder (PID) characterized by a defective host defense against certain bacteria and fungi.1 It is caused by mutations in the CYBB gene, encoding the gp91phox subunit of NADPH oxidase, an enzyme complex responsible for pathogen clearance in professional phagocytic cells. Like many monogenic disorders of the immune system, X-CGD is amenable to gene therapy through gene addition. More recently, scientists have also started to explore nuclease-mediated targeted gene insertion as a strategy to restore gp91phox expression in reprogrammed induced pluripotent stem cells (iPSCs)2–4 and hematopoietic stem and progenitor cells (HSPCs)5 from CGD patients.
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