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Article

Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis

29 May 2014

Abstract excerpt

Regulated transgene expression may improve the safety and efficacy of hematopoietic stem cell (HSC) gene therapy. Clinical trials for X-linked chronic granulomatous disease (X-CGD) employing gammaretroviral vectors were limited by insertional oncogenesis or lack of persistent engraftment. Our novel strategy, based on regulated lentiviral vectors (LV), targets gp91(phox) expression to the differentiated myeloid...

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PMID: 24869932
DOI: 10.1038/mt.2014.87

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