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Article

An assessment of the effects of ectopic gp91phox expression in XCGD iPSC-derived neutrophils

1 Jan 2015

Abstract excerpt

For the treatment of monogenetic hematological disorders, restoration of transgene expression in affected cell populations is generally considered to have beneficial effects. However, X-linked chronic granulomatous disease (XCGD) is unique since the appearance of functional neutrophils in the peripheral blood following hematopoietic stem cell gene therapy is transient only. One contributing factor could be the...

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PMID: 26682238
DOI: 10.1038/mtm.2015.46

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