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Mutations in XLF/NHEJ1/Cernunnos gene results in downregulation of telomerase genes expression and telomere shortening.
Carrillo J, Calvete O, Pintado-Berninches L, Manguan-García C, Sevilla Navarro J, Arias-Salgado EG, Sastre L, Guenechea G, López Granados E, de Villartay JP, Revy P, Benitez J, Perona R. Carrillo J, et al. Among authors: guenechea g. Hum Mol Genet. 2017 May 15;26(10):1900-1914. doi: 10.1093/hmg/ddx098. Hum Mol Genet. 2017. PMID: 28369633
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: guenechea g. 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.
Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.
Holstein M, Mesa-Nuñez C, Miskey C, Almarza E, Poletti V, Schmeer M, Grueso E, Ordóñez Flores JC, Kobelt D, Walther W, Aneja MK, Geiger J, Bonig HB, Izsvák Z, Schleef M, Rudolph C, Mavilio F, Bueren JA, Guenechea G, Ivics Z. Holstein M, et al. Among authors: guenechea g. Mol Ther. 2018 Apr 4;26(4):1137-1153. doi: 10.1016/j.ymthe.2018.01.012. Epub 2018 Jan 31. Mol Ther. 2018. PMID: 29503198 Free PMC article.
Advances in the gene therapy of monogenic blood cell diseases.
Bueren JA, Quintana-Bustamante O, Almarza E, Navarro S, Río P, Segovia JC, Guenechea G. Bueren JA, et al. Among authors: guenechea g. Clin Genet. 2020 Jan;97(1):89-102. doi: 10.1111/cge.13593. Epub 2019 Jul 11. Clin Genet. 2020. PMID: 31231794 Review.
44 results