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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.
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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Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, López-Manzaneda S, Hill C, Del Mar Mañu-Pereira M, Martín MA, Orman I, Vives-Corrons JL, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC.
Garcia-Gomez M, et al. Among authors: lopez manzaneda s.
Mol Ther. 2016 Aug;24(7):1187-98. doi: 10.1038/mt.2016.87. Epub 2016 May 3.
Mol Ther. 2016.
PMID: 27138040
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: lopez manzaneda s.
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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Hematopoietic stem cell transplantation chemotherapy causes microglia senescence and peripheral macrophage engraftment in the brain.
Sailor KA, Agoranos G, López-Manzaneda S, Tada S, Gillet-Legrand B, Guerinot C, Masson JB, Vestergaard CL, Bonner M, Gagnidze K, Veres G, Lledo PM, Cartier N.
Sailor KA, et al. Among authors: lopez manzaneda s.
Nat Med. 2022 Mar;28(3):517-527. doi: 10.1038/s41591-022-01691-9. Epub 2022 Feb 21.
Nat Med. 2022.
PMID: 35190726
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