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Your search keyword '"Leiva, L.E."' showing total 5 results
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5 results on '"Leiva, L.E."'

4. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Author

Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M.P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C.S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J.I., de Saint Basile, G., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L.E., Wissler, M., Prinz, C., Rabbitts, T.H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M.

Subjects
Physiological aspects, Complications and side effects, Research, Gene therapy -- Research -- Physiological aspects, Retroviruses -- Research -- Physiological aspects
Abstract

Ex vivo retrovirus-mediated gene transfer into hematopoietic progenitor cells has been shown to be an efficient strategy to correct inherited diseases of the lymphohematopoietic system, provided that a strong selective [...], We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as γ chain (γc) deficiency] in 9 out of 10 patients by retrovirus-mediated γc gene transfer into autologous CD34 bone marrow cells. However, almost 3 years after gene therapy, uncontrolled exponential clonal proliferation of mature T cells (with γδ+ or αβ+ T cell receptors) has occurred in the two youngest patients. Both patients' clones showed retrovirus vector integration in proximity to the LMO2 proto-oncogene promoter, leading to aberrant transcription and expression of LMO2. Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.

Published
2003

5. Gene Therapy for Severe Combined Immunodeficiency X1.

Author

Hacein-Bey-Abina, S., primary, Schmidt, M., additional, Le Deist, F., additional, Garrigue, A., additional, Borkhardt, A., additional, Wulffraat, N., additional, Alexander, I.E., additional, Wintergerst, U., additional, Leiva, L.E., additional, Delabesse, E., additional, Macintyre, E., additional, Gougeon, M.L., additional, Gyapay, G., additional, Weinssenbach, J., additional, Fraser, C., additional, de Saint-Basile, G., additional, Casanova, J.L., additional, Von Kalle, C., additional, Fischer, A., additional, and Cavazzana-Calvo, M., additional

Published
2005
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