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Page 1
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: gimenez y. 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.
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. Among authors: gimenez y. 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. Among authors: gimenez y. Sci Rep. 2020 Apr 24;10(1):6997. doi: 10.1038/s41598-020-63971-z. Sci Rep. 2020. PMID: 32332829 Free PMC article.
Next-generation Sequencing in Bone Marrow Failure Syndromes and Isolated Cytopenias: Experience of the Spanish Network on Bone Marrow Failure Syndromes.
Gálvez E, Vallespín E, Arias-Salgado EG, Sánchez-Valdepeñas C, Giménez Y, Navarro S, Río P, Bogliolo M, Pujol R, Peiró M, Nevado J, Zubicaray J, Sebastián E, Catalá A, Beléndez C, Díaz de Heredia C, Galera A, Badell I, Madero L, Perona R, Sastre L, Surrallés J, Bueren J, Lapunzina P, Sevilla J. Gálvez E, et al. Among authors: gimenez y. Hemasphere. 2021 Mar 9;5(4):e539. doi: 10.1097/HS9.0000000000000539. eCollection 2021 Apr. Hemasphere. 2021. PMID: 33718801 Free PMC article.
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.
Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia.
Giménez Y, Palacios M, Sánchez-Domínguez R, Zorbas C, Peral J, Puzik A, Ugalde L, Alberquilla O, Villanueva M, Río P, Gálvez E, Da Costa L, Strullu M, Catala A, Ruiz-Llobet A, Segovia JC, Sevilla J, Strahm B, Niemeyer CM, Beléndez C, Leblanc T, Lafontaine DL, Bueren J, Navarro S. Giménez Y, et al. JCI Insight. 2024 May 22;9(10):e171650. doi: 10.1172/jci.insight.171650. JCI Insight. 2024. PMID: 38775150 Free PMC article.
11 results