ZAP-70 restoration in mice by in vivo thymic electroporation.

Viral and non-viral vectors have been developed for gene therapy, but their use is associated with unresolved problems of efficacy and safety. Efficient and safe methods of DNA delivery need to be found for medical application. Here we report a new monopolar system of non-viral electro-gene transfer...

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Autores principales:	Magali Irla, Murielle Saade, Adrien Kissenpfennig, Lionel Franz Poulin, Lee Leserman, Patrice N Marche, Evelyne Jouvin-Marche, François Berger, Catherine Nguyen
Formato:	article
Lenguaje:	EN
Publicado:	Public Library of Science (PLoS) 2008
Materias:	Medicine R Science Q
Acceso en línea:	https://doaj.org/article/ddd9455abee3488286c45c5d97866959
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spelling	oai:doaj.org-article:ddd9455abee3488286c45c5d978669592021-11-25T06:12:38ZZAP-70 restoration in mice by in vivo thymic electroporation.1932-620310.1371/journal.pone.0002059https://doaj.org/article/ddd9455abee3488286c45c5d978669592008-04-01T00:00:00Zhttps://www.ncbi.nlm.nih.gov/pmc/articles/pmid/18446234/?tool=EBIhttps://doaj.org/toc/1932-6203Viral and non-viral vectors have been developed for gene therapy, but their use is associated with unresolved problems of efficacy and safety. Efficient and safe methods of DNA delivery need to be found for medical application. Here we report a new monopolar system of non-viral electro-gene transfer into the thymus in vivo that consists of the local application of electrical pulses after the introduction of the DNA. We assessed the proof of concept of this approach by correcting ZAP-70 deficient severe combined immunodeficiency (SCID) in mice. The thymic electro-gene transfer of the pCMV-ZAP-70-IRES-EGFP vector in these mice resulted in rapid T cell differentiation in the thymus with mature lymphocytes detected by three weeks in secondary lymphoid organs. Moreover, this system resulted in the generation of long-term functional T lymphocytes. Peripheral reconstituted T cells displayed a diversified T cell receptor (TCR) repertoire, and were responsive to alloantigens in vivo. This process applied to the thymus could represent a simplified and effective alternative for gene therapy of T cell immunodeficiencies.Magali IrlaMurielle SaadeAdrien KissenpfennigLionel Franz PoulinLee LesermanPatrice N MarcheEvelyne Jouvin-MarcheFrançois BergerCatherine NguyenPublic Library of Science (PLoS)articleMedicineRScienceQENPLoS ONE, Vol 3, Iss 4, p e2059 (2008)
institution	DOAJ
collection	DOAJ
language	EN
topic	Medicine R Science Q
spellingShingle	Medicine R Science Q Magali Irla Murielle Saade Adrien Kissenpfennig Lionel Franz Poulin Lee Leserman Patrice N Marche Evelyne Jouvin-Marche François Berger Catherine Nguyen ZAP-70 restoration in mice by in vivo thymic electroporation.
description	Viral and non-viral vectors have been developed for gene therapy, but their use is associated with unresolved problems of efficacy and safety. Efficient and safe methods of DNA delivery need to be found for medical application. Here we report a new monopolar system of non-viral electro-gene transfer into the thymus in vivo that consists of the local application of electrical pulses after the introduction of the DNA. We assessed the proof of concept of this approach by correcting ZAP-70 deficient severe combined immunodeficiency (SCID) in mice. The thymic electro-gene transfer of the pCMV-ZAP-70-IRES-EGFP vector in these mice resulted in rapid T cell differentiation in the thymus with mature lymphocytes detected by three weeks in secondary lymphoid organs. Moreover, this system resulted in the generation of long-term functional T lymphocytes. Peripheral reconstituted T cells displayed a diversified T cell receptor (TCR) repertoire, and were responsive to alloantigens in vivo. This process applied to the thymus could represent a simplified and effective alternative for gene therapy of T cell immunodeficiencies.
format	article
author	Magali Irla Murielle Saade Adrien Kissenpfennig Lionel Franz Poulin Lee Leserman Patrice N Marche Evelyne Jouvin-Marche François Berger Catherine Nguyen
author_facet	Magali Irla Murielle Saade Adrien Kissenpfennig Lionel Franz Poulin Lee Leserman Patrice N Marche Evelyne Jouvin-Marche François Berger Catherine Nguyen
author_sort	Magali Irla
title	ZAP-70 restoration in mice by in vivo thymic electroporation.
title_short	ZAP-70 restoration in mice by in vivo thymic electroporation.
title_full	ZAP-70 restoration in mice by in vivo thymic electroporation.
title_fullStr	ZAP-70 restoration in mice by in vivo thymic electroporation.
title_full_unstemmed	ZAP-70 restoration in mice by in vivo thymic electroporation.
title_sort	zap-70 restoration in mice by in vivo thymic electroporation.
publisher	Public Library of Science (PLoS)
publishDate	2008
url	https://doaj.org/article/ddd9455abee3488286c45c5d97866959
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ZAP-70 restoration in mice by in vivo thymic electroporation.

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