Nature Communications (Dec 2018)
CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I
- 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
Affiliations
- Nerea Zabaleta
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Miren Barberia
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Cristina Martin-Higueras
- Hospital Universitario de Canarias, Universidad La Laguna, CIBERER
- Natalia Zapata-Linares
- Regenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Isabel Betancor
- Hospital Universitario de Canarias, Universidad La Laguna, CIBERER
- Saray Rodriguez
- Regenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Rebeca Martinez-Turrillas
- Regenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Laura Torella
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Africa Vales
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Cristina Olagüe
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Amaia Vilas-Zornoza
- Advance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Laura Castro-Labrador
- Advance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- David Lara-Astiaso
- Advance Genomics Laboratory, Oncohematology Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Felipe Prosper
- Regenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Eduardo Salido
- Hospital Universitario de Canarias, Universidad La Laguna, CIBERER
- Gloria Gonzalez-Aseguinolaza
- Gene Therapy and Regulation of Gene Expression Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- Juan R. Rodriguez-Madoz
- Regenerative Medicine Program, Center for Applied Medical Research (CIMA), University of Navarra, IdiSNA
- DOI
- https://doi.org/10.1038/s41467-018-07827-1
- Journal volume & issue
-
Vol. 9,
no. 1
pp. 1 – 9
Abstract
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.
