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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: lopez manzaneda s. 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.
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.
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