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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
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.
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: garcia torralba a.
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.
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Efficient and Fast Generation of Relevant Disease Mouse Models by In Vitro and In Vivo Gene Editing of Zygotes.
Sanchez-Baltasar R, Garcia-Torralba A, Nieto-Romero V, Page A, Molinos-Vicente A, López-Manzaneda S, Ojeda-Pérez I, Ramirez A, Navarro M, Segovia JC, García-Bravo M.
Sanchez-Baltasar R, et al. Among authors: garcia torralba a.
CRISPR J. 2022 Jun;5(3):422-434. doi: 10.1089/crispr.2022.0013.
CRISPR J. 2022.
PMID: 35686982
Free PMC article.
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Helios modulates the maturation of a CA1 neuronal subpopulation required for spatial memory formation.
Giralt A, Brito V, Pardo M, Rubio SE, Marion-Poll L, Martín-Ibáñez R, Zamora-Moratalla A, Bosch C, Ballesteros JJ, Blasco E, García-Torralba A, Pascual M, Pumarola M, Alberch J, Ginés S, Martín ED, Segovia J, Soriano E, Canals JM.
Giralt A, et al. Among authors: garcia torralba a.
Exp Neurol. 2020 Jan;323:113095. doi: 10.1016/j.expneurol.2019.113095. Epub 2019 Nov 8.
Exp Neurol. 2020.
PMID: 31712124
Free 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: garcia torralba 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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