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.
Bibliographic Details
| Main Authors: |
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 |
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| Format: | Article
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| Language: | English |
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| Published: |
Nature Publishing Group
2018-12-01
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| Series: | Nature Communications
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| Online Access: | https://doi.org/10.1038/s41467-018-07827-1
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