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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Nature Publishing Group
2018-12-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/s41467-018-07827-1 |
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doaj-4b179c1567b1437ea464d69ab6bf54552021-05-11T10:27:16ZengNature Publishing GroupNature Communications2041-17232018-12-01911910.1038/s41467-018-07827-1CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type INerea Zabaleta0Miren Barberia1Cristina Martin-Higueras2Natalia Zapata-Linares3Isabel Betancor4Saray Rodriguez5Rebeca Martinez-Turrillas6Laura Torella7Africa Vales8Cristina Olagüe9Amaia Vilas-Zornoza10Laura Castro-Labrador11David Lara-Astiaso12Felipe Prosper13Eduardo Salido14Gloria Gonzalez-Aseguinolaza15Juan R. Rodriguez-Madoz16Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAGene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAHospital Universitario de Canarias, Universidad La Laguna, CIBERERRegenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAHospital Universitario de Canarias, Universidad La Laguna, CIBERERRegenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNARegenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAGene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAGene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAGene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAAdvance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAAdvance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAAdvance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNARegenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNAHospital Universitario de Canarias, Universidad La Laguna, CIBERERGene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNARegenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNASubstrate 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.https://doi.org/10.1038/s41467-018-07827-1 |
| collection |
DOAJ |
| language |
English |
| format |
Article |
| sources |
DOAJ |
| 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 |
| spellingShingle |
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 Nature Communications |
| 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 Publishing Group |
| series |
Nature Communications |
| issn |
2041-1723 |
| publishDate |
2018-12-01 |
| 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. |
| url |
https://doi.org/10.1038/s41467-018-07827-1 |
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