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Novel vectors and approaches for gene therapy in liver diseases.
Maestro S, Weber ND, Zabaleta N, Aldabe R, Gonzalez-Aseguinolaza G. Maestro S, et al. Among authors: zabaleta n. JHEP Rep. 2021 Apr 30;3(4):100300. doi: 10.1016/j.jhepr.2021.100300. eCollection 2021 Aug. JHEP Rep. 2021. PMID: 34159305 Free PMC article. Review.
CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I.
Zabaleta N, Barberia M, Martin-Higueras C, Zapata-Linares N, Betancor I, Rodriguez S, Martinez-Turrillas R, Torella L, Vales A, Olagüe C, Vilas-Zornoza A, Castro-Labrador L, Lara-Astiaso D, Prosper F, Salido E, Gonzalez-Aseguinolaza G, Rodriguez-Madoz JR. Zabaleta N, et al. Nat Commun. 2018 Dec 21;9(1):5454. doi: 10.1038/s41467-018-07827-1. Nat Commun. 2018. PMID: 30575740 Free PMC article.
In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9.
López-Manzaneda S, Ojeda-Pérez I, Zabaleta N, García-Torralba A, Alberquilla O, Torres R, Sánchez-Domínguez R, Torella L, Olivier E, Mountford J, Ramírez JC, Bueren JA, González-Aseguinolaza G, Segovia JC. López-Manzaneda S, et al. Among authors: zabaleta n. Mol Ther Methods Clin Dev. 2020 Oct 15;19:426-437. doi: 10.1016/j.omtm.2020.10.007. eCollection 2020 Dec 11. Mol Ther Methods Clin Dev. 2020. PMID: 33294491 Free PMC article.
Gene therapy for liver diseases - progress and challenges.
Zabaleta N, Unzu C, Weber ND, Gonzalez-Aseguinolaza G. Zabaleta N, et al. Nat Rev Gastroenterol Hepatol. 2023 May;20(5):288-305. doi: 10.1038/s41575-022-00729-0. Epub 2023 Jan 16. Nat Rev Gastroenterol Hepatol. 2023. PMID: 36646909 Review.
27 results