Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevents disease progression in a mouse disease model.

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Autores principales:	Nicholas D. Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2019
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/01e5e4ee2f0f4ae9875e80c35af9e27b
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spelling	oai:doaj.org-article:01e5e4ee2f0f4ae9875e80c35af9e27b2021-12-02T16:57:33ZGene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model10.1038/s41467-019-13614-32041-1723https://doaj.org/article/01e5e4ee2f0f4ae9875e80c35af9e27b2019-12-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-13614-3https://doaj.org/toc/2041-1723Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevents disease progression in a mouse disease model.Nicholas D. WeberLeticia OdriozolaJavier Martínez-GarcíaVeronica FerrerAnne DouarBernard BénichouGloria González-AseguinolazaCristian SmerdouNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-13 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Nicholas D. Weber Leticia Odriozola Javier Martínez-García Veronica Ferrer Anne Douar Bernard Bénichou Gloria González-Aseguinolaza Cristian Smerdou Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
description	Progressive familial intrahepatic cholestasis type 3 is a disease caused by mutations in the gene encoding the multidrug resistance protein 3, and has limited treatment options. Here they show that adeno-associated virus mediated gene therapy prevents disease progression in a mouse disease model.
format	article
author	Nicholas D. Weber Leticia Odriozola Javier Martínez-García Veronica Ferrer Anne Douar Bernard Bénichou Gloria González-Aseguinolaza Cristian Smerdou
author_facet	Nicholas D. Weber Leticia Odriozola Javier Martínez-García Veronica Ferrer Anne Douar Bernard Bénichou Gloria González-Aseguinolaza Cristian Smerdou
author_sort	Nicholas D. Weber
title	Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
title_short	Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
title_full	Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
title_fullStr	Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
title_full_unstemmed	Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
title_sort	gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/01e5e4ee2f0f4ae9875e80c35af9e27b
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Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

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