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Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
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.
Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.
Quintana-Bustamante O, Fañanas-Baquero S, Orman I, Torres R, Duchateau P, Poirot L, Gouble A, Bueren JA, Segovia JC.
Quintana-Bustamante O, et al. Among authors: fananas baquero s.
PLoS One. 2019 Oct 16;14(10):e0223775. doi: 10.1371/journal.pone.0223775. eCollection 2019.
PLoS One. 2019.
PMID: 31618280
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.
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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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: fananas baquero 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.
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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: fananas baquero s.
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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Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice.
Fañanas-Baquero S, Orman I, Becerra Aparicio F, Bermudez de Miguel S, Garcia Merino J, Yañez R, Fernandez Sainz Y, Sánchez R, Dessy-Rodríguez M, Alberquilla O, Alfaro D, Zapata A, Bueren JA, Segovia JC, Quintana-Bustamante O.
Fañanas-Baquero S, et al.
Haematologica. 2021 Jun 1;106(6):1659-1670. doi: 10.3324/haematol.2019.233924.
Haematologica. 2021.
PMID: 32354868
Free PMC article.
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