Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

Hereditary tyrosinaemia type I is caused by a gene defect that leads to a lethal accumulation of toxic metabolites in the liver. Here the authors use CRISPR/Cas9 to 'cure' the disease in mice by inactivating another gene, rather than targeting the disease-causing gene itself, to reroute he...

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Autores principales:	Francis P. Pankowicz, Mercedes Barzi, Xavier Legras, Leroy Hubert, Tian Mi, Julie A. Tomolonis, Milan Ravishankar, Qin Sun, Diane Yang, Malgorzata Borowiak, Pavel Sumazin, Sarah H. Elsea, Beatrice Bissig-Choisat, Karl-Dimiter Bissig
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2016
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d
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spelling	oai:doaj.org-article:c9a870f56fb24cb1bba81c0bca196a4d2021-12-02T14:39:29ZReprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia10.1038/ncomms126422041-1723https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d2016-08-01T00:00:00Zhttps://doi.org/10.1038/ncomms12642https://doaj.org/toc/2041-1723Hereditary tyrosinaemia type I is caused by a gene defect that leads to a lethal accumulation of toxic metabolites in the liver. Here the authors use CRISPR/Cas9 to 'cure' the disease in mice by inactivating another gene, rather than targeting the disease-causing gene itself, to reroute hepatic tyrosine catabolism.Francis P. PankowiczMercedes BarziXavier LegrasLeroy HubertTian MiJulie A. TomolonisMilan RavishankarQin SunDiane YangMalgorzata BorowiakPavel SumazinSarah H. ElseaBeatrice Bissig-ChoisatKarl-Dimiter BissigNature PortfolioarticleScienceQENNature Communications, Vol 7, Iss 1, Pp 1-6 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Francis P. Pankowicz Mercedes Barzi Xavier Legras Leroy Hubert Tian Mi Julie A. Tomolonis Milan Ravishankar Qin Sun Diane Yang Malgorzata Borowiak Pavel Sumazin Sarah H. Elsea Beatrice Bissig-Choisat Karl-Dimiter Bissig Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
description	Hereditary tyrosinaemia type I is caused by a gene defect that leads to a lethal accumulation of toxic metabolites in the liver. Here the authors use CRISPR/Cas9 to 'cure' the disease in mice by inactivating another gene, rather than targeting the disease-causing gene itself, to reroute hepatic tyrosine catabolism.
format	article
author	Francis P. Pankowicz Mercedes Barzi Xavier Legras Leroy Hubert Tian Mi Julie A. Tomolonis Milan Ravishankar Qin Sun Diane Yang Malgorzata Borowiak Pavel Sumazin Sarah H. Elsea Beatrice Bissig-Choisat Karl-Dimiter Bissig
author_facet	Francis P. Pankowicz Mercedes Barzi Xavier Legras Leroy Hubert Tian Mi Julie A. Tomolonis Milan Ravishankar Qin Sun Diane Yang Malgorzata Borowiak Pavel Sumazin Sarah H. Elsea Beatrice Bissig-Choisat Karl-Dimiter Bissig
author_sort	Francis P. Pankowicz
title	Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
title_short	Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
title_full	Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
title_fullStr	Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
title_full_unstemmed	Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia
title_sort	reprogramming metabolic pathways in vivo with crispr/cas9 genome editing to treat hereditary tyrosinaemia
publisher	Nature Portfolio
publishDate	2016
url	https://doaj.org/article/c9a870f56fb24cb1bba81c0bca196a4d
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Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

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