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research article

Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease

Barde, I.
•
Laurenti, E.
•
Verp, S.
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2011
Gene therapy

Insertional mutagenesis represents a serious adverse effect of gene therapy with integrating vectors. However, although uncontrolled activation of growth-promoting genes in stem cells can predictably lead to oncological processes, this is far less likely if vector transcriptional activity can be restricted to fully differentiated cells. Diseases requiring phenotypic correction only in mature cells offer such an opportunity, provided that lineage/stage-restricted systems can be properly tailored. In this study, we followed this reasoning to design lentiviral vectors for the gene therapy of chronic granulomatous disease (CGD), an immune deficiency due a loss of nicotinamide adenine dinucleotide phosphate (NADPH) oxidase in phagocytes, most often secondary to mutations in gp91(phox). Using self-inactivating HIV1-derived vectors as background, we first expressed enhanced green fluorescent protein (eGFP) from a minimal gp91(phox) promoter, adding various natural or synthetic transcriptional regulatory elements to foster both specificity and potency. The resulting vectors were assessed either by transplantation or by lentiviral transgenesis, searching for combinations conferring strong and specific expression into mature phagocytic cells. The most promising vector was modified to express gp91(phox) and used to treat CGD mice. High-level restoration of NADPH activity was documented in granulocytes from the treated animals. We propose that this lineage-specific lentiviral vector is a suitable candidate for the gene therapy of CGD.

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Type
research article
DOI
10.1038/gt.2011.65
Web of Science ID

WOS:000296889500008

Author(s)
Barde, I.
Laurenti, E.
Verp, S.
Wiznerowicz, M.
Offner, S.
Viornery, A.
Galy, A.
Trumpp, A.
Trono, D.  
Date Issued

2011

Published in
Gene therapy
Volume

18

Issue

11

Start page

1087

End page

97

Subjects

Genetic Vectors

Editorial or Peer reviewed

NON-REVIEWED

Written at

EPFL

EPFL units
LVG  
Available on Infoscience
May 6, 2011
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/67136
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