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Page 1
Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis.
Fernandez-Perez R, Lopez-Santalla M, Sánchez-Domínguez R, Alberquilla O, Gutiérrez-Cañas I, Juarranz Y, Bueren JA, Garin MI. Fernandez-Perez R, et al. Among authors: sanchez dominguez r. Front Immunol. 2021 Jun 28;12:687443. doi: 10.3389/fimmu.2021.687443. eCollection 2021. Front Immunol. 2021. PMID: 34262567 Free PMC article.
Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients.
Río P, Navarro S, Guenechea G, Sánchez-Domínguez R, Lamana ML, Yañez R, Casado JA, Mehta PA, Pujol MR, Surrallés J, Charrier S, Galy A, Segovia JC, Díaz de Heredia C, Sevilla J, Bueren JA. Río P, et al. Blood. 2017 Sep 28;130(13):1535-1542. doi: 10.1182/blood-2017-03-774174. Epub 2017 Aug 11. Blood. 2017. PMID: 28801449 Free article.
Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia.
Río P, Navarro S, Wang W, Sánchez-Domínguez R, Pujol RM, Segovia JC, Bogliolo M, Merino E, Wu N, Salgado R, Lamana ML, Yañez RM, Casado JA, Giménez Y, Román-Rodríguez FJ, Álvarez L, Alberquilla O, Raimbault A, Guenechea G, Lozano ML, Cerrato L, Hernando M, Gálvez E, Hladun R, Giralt I, Barquinero J, Galy A, García de Andoín N, López R, Catalá A, Schwartz JD, Surrallés J, Soulier J, Schmidt M, Díaz de Heredia C, Sevilla J, Bueren JA. Río P, et al. Nat Med. 2019 Sep;25(9):1396-1401. doi: 10.1038/s41591-019-0550-z. Epub 2019 Sep 9. Nat Med. 2019. PMID: 31501599 Clinical Trial.
TALEN mediated gene editing in a mouse model of Fanconi anemia.
Pino-Barrio MJ, Giménez Y, Villanueva M, Hildenbeutel M, Sánchez-Dominguez R, Rodríguez-Perales S, Pujol R, Surrallés J, Río P, Cathomen T, Mussolino C, Bueren JA, Navarro S. Pino-Barrio MJ, et al. Sci Rep. 2020 Apr 24;10(1):6997. doi: 10.1038/s41598-020-63971-z. Sci Rep. 2020. PMID: 32332829 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.
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes.
Sevilla J, Navarro S, Rio P, Sánchez-Domínguez R, Zubicaray J, Gálvez E, Merino E, Sebastián E, Azqueta C, Casado JA, Segovia JC, Alberquilla O, Bogliolo M, Román-Rodríguez FJ, Giménez Y, Larcher L, Salgado R, Pujol RM, Hladun R, Castillo A, Soulier J, Querol S, Fernández J, Schwartz J, García de Andoín N, López R, Catalá A, Surralles J, Díaz-de-Heredia C, Bueren JA. Sevilla J, et al. Among authors: sanchez dominguez r. Mol Ther Methods Clin Dev. 2021 Jun 12;22:66-75. doi: 10.1016/j.omtm.2021.06.001. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485595 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. Among authors: sanchez dominguez r. 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: sanchez dominguez r. 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.
21 results