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Efficient and Fast Generation of Relevant Disease Mouse Models by In Vitro and In Vivo Gene Editing of Zygotes.
CRISPR J. 2022 Jun;5(3):422-434. doi: 10.1089/crispr.2022.0013.
CRISPR J. 2022.
PMID: 35686982
Free PMC article.
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Navarro S, Quintana-Bustamante O, Sanchez-Dominguez R, Lopez-Manzaneda S, Ojeda-Perez I, Garcia-Torralba A, Alberquilla O, Law K, Beard BC, Bastone A, Rothe M, Villanueva M, Ramirez JC, Fañanas-Baquero S, Nieto-Romero V, Molinos-Vicente A, Gutierrez S, Nicoletti E, García-Bravo M, Bueren JA, Schwartz JD, Segovia JC.
Navarro S, et al. Among authors: molinos vicente a.
Mol Ther Methods Clin Dev. 2021 Jul 29;22:350-359. doi: 10.1016/j.omtm.2021.07.006. eCollection 2021 Sep 10.
Mol Ther Methods Clin Dev. 2021.
PMID: 34514027
Free PMC article.
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Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts.
Nieto-Romero V, García-Torralba A, Molinos-Vicente A, Moya FJ, Rodríguez-Perales S, García-Escudero R, Salido E, Segovia JC, García-Bravo M.
Nieto-Romero V, et al. Among authors: molinos vicente a.
iScience. 2024 Mar 21;27(4):109530. doi: 10.1016/j.isci.2024.109530. eCollection 2024 Apr 19.
iScience. 2024.
PMID: 38577102
Free PMC article.
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