CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

Substrate reduction therapies (SRT) are a promising therapeutic approach for monogenic inherited metabolic diseases. Here the authors evaluate the therapeutic potential of an in vivo CRISPR/Cas9-mediated SRT to treat primary hyperoxaluria type I and demonstrate its safety and efficacy.

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Autores principales:	Nerea Zabaleta, Miren Barberia, Cristina Martin-Higueras, Natalia Zapata-Linares, Isabel Betancor, Saray Rodriguez, Rebeca Martinez-Turrillas, Laura Torella, Africa Vales, Cristina Olagüe, Amaia Vilas-Zornoza, Laura Castro-Labrador, David Lara-Astiaso, Felipe Prosper, Eduardo Salido, Gloria Gonzalez-Aseguinolaza, Juan R. Rodriguez-Madoz
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2018
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/4b179c1567b1437ea464d69ab6bf5455
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spelling	oai:doaj.org-article:4b179c1567b1437ea464d69ab6bf54552021-12-02T14:38:53ZCRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I10.1038/s41467-018-07827-12041-1723https://doaj.org/article/4b179c1567b1437ea464d69ab6bf54552018-12-01T00:00:00Zhttps://doi.org/10.1038/s41467-018-07827-1https://doaj.org/toc/2041-1723Substrate reduction therapies (SRT) are a promising therapeutic approach for monogenic inherited metabolic diseases. Here the authors evaluate the therapeutic potential of an in vivo CRISPR/Cas9-mediated SRT to treat primary hyperoxaluria type I and demonstrate its safety and efficacy.Nerea ZabaletaMiren BarberiaCristina Martin-HiguerasNatalia Zapata-LinaresIsabel BetancorSaray RodriguezRebeca Martinez-TurrillasLaura TorellaAfrica ValesCristina OlagüeAmaia Vilas-ZornozaLaura Castro-LabradorDavid Lara-AstiasoFelipe ProsperEduardo SalidoGloria Gonzalez-AseguinolazaJuan R. Rodriguez-MadozNature PortfolioarticleScienceQENNature Communications, Vol 9, Iss 1, Pp 1-9 (2018)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Nerea Zabaleta Miren Barberia Cristina Martin-Higueras Natalia Zapata-Linares Isabel Betancor Saray Rodriguez Rebeca Martinez-Turrillas Laura Torella Africa Vales Cristina Olagüe Amaia Vilas-Zornoza Laura Castro-Labrador David Lara-Astiaso Felipe Prosper Eduardo Salido Gloria Gonzalez-Aseguinolaza Juan R. Rodriguez-Madoz CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
description	Substrate reduction therapies (SRT) are a promising therapeutic approach for monogenic inherited metabolic diseases. Here the authors evaluate the therapeutic potential of an in vivo CRISPR/Cas9-mediated SRT to treat primary hyperoxaluria type I and demonstrate its safety and efficacy.
format	article
author	Nerea Zabaleta Miren Barberia Cristina Martin-Higueras Natalia Zapata-Linares Isabel Betancor Saray Rodriguez Rebeca Martinez-Turrillas Laura Torella Africa Vales Cristina Olagüe Amaia Vilas-Zornoza Laura Castro-Labrador David Lara-Astiaso Felipe Prosper Eduardo Salido Gloria Gonzalez-Aseguinolaza Juan R. Rodriguez-Madoz
author_facet	Nerea Zabaleta Miren Barberia Cristina Martin-Higueras Natalia Zapata-Linares Isabel Betancor Saray Rodriguez Rebeca Martinez-Turrillas Laura Torella Africa Vales Cristina Olagüe Amaia Vilas-Zornoza Laura Castro-Labrador David Lara-Astiaso Felipe Prosper Eduardo Salido Gloria Gonzalez-Aseguinolaza Juan R. Rodriguez-Madoz
author_sort	Nerea Zabaleta
title	CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
title_short	CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
title_full	CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
title_fullStr	CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
title_full_unstemmed	CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
title_sort	crispr/cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type i
publisher	Nature Portfolio
publishDate	2018
url	https://doaj.org/article/4b179c1567b1437ea464d69ab6bf5455
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CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

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