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High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease.
Murillo O, Collantes M, Gazquez C, Moreno D, Hernandez-Alcoceba R, Barberia M, Ecay M, Tamarit B, Douar A, Ferrer V, Combal JP, Peñuelas I, Bénichou B, Gonzalez-Aseguinolaza G. Murillo O, et al. Among authors: barberia m. Mol Ther Methods Clin Dev. 2022 Jun 9;26:98-106. doi: 10.1016/j.omtm.2022.06.001. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35795774 Free PMC article.
Liver Expression of a MiniATP7B Gene Results in Long-Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice.
Murillo O, Moreno D, Gazquez C, Barberia M, Cenzano I, Navarro I, Uriarte I, Sebastian V, Arruebo M, Ferrer V, Bénichou B, Combal JP, Prieto J, Hernandez-Alcoceba R, Gonzalez Aseguinolaza G. Murillo O, et al. Among authors: barberia m. Hepatology. 2019 Jul;70(1):108-126. doi: 10.1002/hep.30535. Epub 2019 Mar 20. Hepatology. 2019. PMID: 30706949
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. Among authors: barberia m. Nat Commun. 2018 Dec 21;9(1):5454. doi: 10.1038/s41467-018-07827-1. Nat Commun. 2018. PMID: 30575740 Free PMC article.