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Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.
García M, Bonafont J, Martínez-Palacios J, Xu R, Turchiano G, Svensson S, Thrasher AJ, Larcher F, Del Rio M, Hernández-Alcoceba R, Garín MI, Mencía Á, Murillas R. García M, et al. Among authors: hernandez alcoceba r. Mol Ther Methods Clin Dev. 2022 Sep 16;27:96-108. doi: 10.1016/j.omtm.2022.09.005. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36212909 Free PMC article.
Gene supplementation of CYP27A1 in the liver restores bile acid metabolism in a mouse model of cerebrotendinous xanthomatosis.
Lumbreras S, Ricobaraza A, Baila-Rueda L, Gonzalez-Aparicio M, Mora-Jimenez L, Uriarte I, Bunuales M, Avila MA, Monte MJ, Marin JJG, Cenarro A, Gonzalez-Aseguinolaza G, Hernandez-Alcoceba R. Lumbreras S, et al. Mol Ther Methods Clin Dev. 2021 Jul 21;22:210-221. doi: 10.1016/j.omtm.2021.07.002. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485606 Free PMC article.
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: hernandez alcoceba r. 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.
Short-Term Local Expression of a PD-L1 Blocking Antibody from a Self-Replicating RNA Vector Induces Potent Antitumor Responses.
Ballesteros-Briones MC, Martisova E, Casales E, Silva-Pilipich N, Buñuales M, Galindo J, Mancheño U, Gorraiz M, Lasarte JJ, Kochan G, Escors D, Sanchez-Paulete AR, Melero I, Prieto J, Hernandez-Alcoceba R, Hervas-Stubbs S, Smerdou C. Ballesteros-Briones MC, et al. Mol Ther. 2019 Nov 6;27(11):1892-1905. doi: 10.1016/j.ymthe.2019.09.016. Epub 2019 Sep 16. Mol Ther. 2019. PMID: 31563534 Free PMC article.
Recombinant adenoviral vectors turn on the type I interferon system without inhibition of transgene expression and viral replication.
Huarte E, Larrea E, Hernández-Alcoceba R, Alfaro C, Murillo O, Arina A, Tirapu I, Azpilicueta A, Hervás-Stubbs S, Bortolanza S, Pérez-Gracia JL, Civeira MP, Prieto J, Riezu-Boj JI, Melero I. Huarte E, et al. Mol Ther. 2006 Jul;14(1):129-38. doi: 10.1016/j.ymthe.2006.02.015. Epub 2006 Apr 19. Mol Ther. 2006. PMID: 16627004 Free article.
Danio Rerio as Model Organism for Adenoviral Vector Evaluation.
Gulías P, Guerra-Varela J, Gonzalez-Aparicio M, Ricobaraza A, Vales A, Gonzalez-Aseguinolaza G, Hernandez-Alcoceba R, Sánchez L. Gulías P, et al. Genes (Basel). 2019 Dec 17;10(12):1053. doi: 10.3390/genes10121053. Genes (Basel). 2019. PMID: 31861246 Free PMC article.
84 results