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Article

Gene therapy for chronic granulomatous disease

1 Sept 2004

Abstract excerpt

Chronic granulomatous disease (CGD) is a congenital immune deficiency that is a promising therapeutic target for gene replacement into haematopoietic stem cells (HSCs). CGD results from mutations in any one of four genes encoding subunits of the superoxide-generating NADPH oxidase of phagocytes. Life-threatening, recurrent bacterial and fungal infections, as well as inflammatory granulomas, are the hallmarks of...

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Identifiers

PMID: 15335310
DOI: 10.1517/14712598.4.9.1423

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