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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. 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.
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.
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.
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.
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.