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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: ojeda perez i.
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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Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, Sanchez-Dominguez R, Camarena J, Ojeda-Perez I, Dessy-Rodriguez M, Turk R, Schubert MS, Lattanzi A, Xu L, Lopez-Lorenzo JL, Bianchi P, Bueren JA, Behlke MA, Porteus M, Segovia JC.
Fañanas-Baquero S, et al. Among authors: ojeda perez i.
Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10.
Mol Ther Methods Clin Dev. 2021.
PMID: 34485608
Free PMC article.
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Gene Editing for Inherited Red Blood Cell Diseases.
Quintana-Bustamante O, Fañanas-Baquero S, Dessy-Rodriguez M, Ojeda-Pérez I, Segovia JC.
Quintana-Bustamante O, et al. Among authors: ojeda perez i.
Front Physiol. 2022 Mar 28;13:848261. doi: 10.3389/fphys.2022.848261. eCollection 2022.
Front Physiol. 2022.
PMID: 35418876
Free PMC article.
Review.
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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: ojeda perez i.
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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ERAS, a Member of the Ras Superfamily, Acts as an Oncoprotein in the Mammary Gland.
Suarez-Cabrera C, Ojeda-Perez I, Sanchez-Baltasar R, Page A, Bravo A, Navarro M, Ramirez A.
Suarez-Cabrera C, et al. Among authors: ojeda perez i.
Cancers (Basel). 2021 Nov 8;13(21):5588. doi: 10.3390/cancers13215588.
Cancers (Basel). 2021.
PMID: 34771750
Free PMC article.
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Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
Fañanas-Baquero S, Morín M, Fernández S, Ojeda-Perez I, Dessy-Rodriguez M, Giurgiu M, Bueren JA, Moreno-Pelayo MA, Segovia JC, Quintana-Bustamante O.
Fañanas-Baquero S, et al. Among authors: ojeda perez i.
Front Genome Ed. 2023 Apr 28;5:1104666. doi: 10.3389/fgeed.2023.1104666. eCollection 2023.
Front Genome Ed. 2023.
PMID: 37188156
Free PMC article.
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