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Page 1
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: roman rodriguez fj. 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.
Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients.
Diez B, Genovese P, Roman-Rodriguez FJ, Alvarez L, Schiroli G, Ugalde L, Rodriguez-Perales S, Sevilla J, Diaz de Heredia C, Holmes MC, Lombardo A, Naldini L, Bueren JA, Rio P. Diez B, et al. Among authors: roman rodriguez fj. EMBO Mol Med. 2017 Nov;9(11):1574-1588. doi: 10.15252/emmm.201707540. EMBO Mol Med. 2017. PMID: 28899930 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: roman rodriguez fj. 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.
NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia.
Román-Rodríguez FJ, Ugalde L, Álvarez L, Díez B, Ramírez MJ, Risueño C, Cortón M, Bogliolo M, Bernal S, March F, Ayuso C, Hanenberg H, Sevilla J, Rodríguez-Perales S, Torres-Ruiz R, Surrallés J, Bueren JA, Río P. Román-Rodríguez FJ, et al. Cell Stem Cell. 2019 Nov 7;25(5):607-621.e7. doi: 10.1016/j.stem.2019.08.016. Epub 2019 Sep 19. Cell Stem Cell. 2019. PMID: 31543367 Free article.
Generating New FANCA-Deficient HNSCC Cell Lines by Genomic Editing Recapitulates the Cellular Phenotypes of Fanconi Anemia.
Errazquin R, Sieiro E, Moreno P, Ramirez MJ, Lorz C, Peral J, Ortiz J, Casado JA, Roman-Rodriguez FJ, Hanenberg H, Río P, Surralles J, Segrelles C, Garcia-Escudero R. Errazquin R, et al. Among authors: roman rodriguez fj. Genes (Basel). 2021 Apr 9;12(4):548. doi: 10.3390/genes12040548. Genes (Basel). 2021. PMID: 33918752 Free PMC article.
Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent.
Lévy C, Amirache F, Girard-Gagnepain A, Frecha C, Roman-Rodríguez FJ, Bernadin O, Costa C, Nègre D, Gutierrez-Guerrero A, Vranckx LS, Clerc I, Taylor N, Thielecke L, Cornils K, Bueren JA, Rio P, Gijsbers R, Cosset FL, Verhoeyen E. Lévy C, et al. Among authors: roman rodriguez fj. Blood Adv. 2017 Oct 24;1(23):2088-2104. doi: 10.1182/bloodadvances.2017007773. eCollection 2017 Oct 24. Blood Adv. 2017. PMID: 29296856 Free PMC article.
Involvement of DNMT3B in the pathogenesis of Hirschsprung disease and its possible role as a regulator of neurogenesis in the human enteric nervous system.
Torroglosa A, Enguix-Riego MV, Fernández RM, Román-Rodriguez FJ, Moya-Jiménez MJ, de Agustín JC, Antiñolo G, Borrego S. Torroglosa A, et al. Among authors: roman rodriguez fj. Genet Med. 2014 Sep;16(9):703-10. doi: 10.1038/gim.2014.17. Epub 2014 Feb 27. Genet Med. 2014. PMID: 24577265 Free article.
Waardenburg syndrome type 4: report of two new cases caused by SOX10 mutations in Spain.
Fernández RM, Núñez-Ramos R, Enguix-Riego MV, Román-Rodríguez FJ, Galán-Gómez E, Blesa-Sánchez E, Antiñolo G, Núñez-Núñez R, Borrego S. Fernández RM, et al. Among authors: roman rodriguez fj. Am J Med Genet A. 2014 Feb;164A(2):542-7. doi: 10.1002/ajmg.a.36302. Epub 2013 Dec 5. Am J Med Genet A. 2014. PMID: 24311220
Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.
Ellison S, Buckland K, Learmonth Y, Day V, Kalra S, Howe L, Roman-Rodriguez FJ, Bonafont J, Booth L, Holley R, Smythe J, Jones S, Thrasher A, Booth C, Bigger BW. Ellison S, et al. Among authors: roman rodriguez fj. Mol Ther Methods Clin Dev. 2024 May 21;32(2):101271. doi: 10.1016/j.omtm.2024.101271. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38946936 Free PMC article.