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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients

Charrier, S, Dupré, L, Scaramuzza, S, Jeanson-Leh, L, Blundell, M P, Danos, O, Cattaneo, F, Aiuti, A, Eckenberg, R, Thrasher, A J, Roncarolo, M Grazia, Galy, A·2007·Gene Therapy
DOI 10.1038/sj.gt.3302863
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Abstract

Gene therapy has been proposed as a potential treatment for Wiskott-Aldrich syndrome (WAS), a severe primary immune deficiency characterized by multiple hematopoietic-specific cellular defects. In order to develop an optimal lentiviral gene transfer cassette for this application, we compared the performance of several internal promoters in a variety of cell lineages from human WAS patients. Vectors using endogenous promoters derived from short (0.5 kb) or long (1.6 kb) 5' flanking sequences of the WAS gene, expressed the transgene in T, B, dendritic cells as well as CD34(+) progenitor cells, but functioned poorly in non-hematopoietic cells. Defects of T-cell proliferation and interleukin-2 production, and the cytoskeletal anomalies in WAS dendritic cells were also corrected. The levels of reconstitution were comparable to those obtained following transduction with similar lentiviral vectors incorporating constitutive PGK-1, EF1-alpha promoters or the spleen focus forming virus gammaretroviral LTR. Thus, native regulatory sequences target the expression of the therapeutic WAS transgene to the hematopoietic system, as is naturally the case for WAS, and are effective for correction of multiple cellular defects. These vectors may have significant advantages for clinical application in terms of natural gene regulation, and reduction in the potential for adverse mutagenic events.

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Research works printed but not linked: 34

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Cited by

Metadata

Authors
Charrier, S., Dupré, L., Scaramuzza, S., Jeanson-Leh, L., Blundell, M. P., Danos, O., Cattaneo, F., Aiuti, A., Eckenberg, R., Thrasher, A. J., Roncarolo, M. G., & Galy, A.
Venue
Gene Therapy
Publisher
Springer Science and Business Media LLC
Published
2006-10-19
Volume
14
Issue
5
Pages
415-428
ISSN
  • 0969-7128
  • 1476-5462
DOI
10.1038/sj.gt.3302863
Cited by
91
References
0
Language
en
Licence

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