Lentiviral gene therapy rescues p47^phox chronic granulomatous disease and the ability to fight Salmonella infection in mice.

ABSTRACT: Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency disorder characterised by recurrent and often life-threatening infections and hyperinflammation. It is caused by defects of the phagocytic NADPH oxidase, a multicomponent enzyme system responsible for effective pathogen killing. A phase I/II clinical trial of lentiviral gene therapy is underway for the most common form of CGD, X-linked, caused by mutations in the gp91^phox subunit of the NADPH oxidase. We propose to use a similar strategy to tackle p47^phox-deficient CGD, caused by mutations in NCF1, which encodes the p47^phox cytosolic component of the enzymatic complex. We generated a pCCLCHIM-p47^phox lentiviral vector, containing the chimeric Cathepsin G/FES myeloid prom

SUBMITTER: Schejtman A 

PROVIDER: S-EPMC7500983 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature