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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D, Arechavala-Gomeza V, Peng L, Hapil FZ, Harvey J, Ortega FG, Gonzalez-Martinez C, Lederer CW, Mikkelsen K, Gasiunas G, Kalter N, Gonçalves MAFV, Petersen J, Garanto A, Montoliu L, Maresca M, Seemann SE, Gorodkin J, Mazini L, Sanchez R, Rodriguez-Madoz JR, Maldonado-Pérez N, Laura T, Schmueck-Henneresse M, Maccalli C, Grünewald J, Carmona G, Kachamakova-Trojanowska N, Miccio A, Martin F, Turchiano G, Cathomen T, Luo Y, Tsai SQ, Benabdellah K; COST Action CA21113. Cavazza A, et al. Among authors: rio p. Mol Ther Nucleic Acids. 2023 Oct 29;34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38034032 Free PMC article. Review.
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: rio p. Sci Rep. 2020 Apr 24;10(1):6997. doi: 10.1038/s41598-020-63971-z. Sci Rep. 2020. PMID: 32332829 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: rio p. Clin Genet. 2020 Jan;97(1):89-102. doi: 10.1111/cge.13593. Epub 2019 Jul 11. Clin Genet. 2020. PMID: 31231794 Review.
CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative.
Luque J, Mendes I, Gómez B, Morte B, López de Heredia M, Herreras E, Corrochano V, Bueren J, Gallano P, Artuch R, Fillat C, Pérez-Jurado LA, Montoliu L, Carracedo Á, Millán JM, Webb SM, Palau F; CIBERER Network; Lapunzina P. Luque J, et al. Clin Genet. 2022 May;101(5-6):481-493. doi: 10.1111/cge.14113. Epub 2022 Feb 4. Clin Genet. 2022. PMID: 35060122 Free PMC article. Review.
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I.
Mesa-Núñez C, Damián C, Fernández-García M, Díez B, Rao G, Schwartz JD, Law KM, Sevilla J, Río P, Yáñez R, Bueren JA, Almarza E. Mesa-Núñez C, et al. Among authors: rio p. Mol Ther Methods Clin Dev. 2022 Aug 1;26:459-470. doi: 10.1016/j.omtm.2022.07.015. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092365 Free PMC article.
Advances in Gene Therapy for Fanconi Anemia.
Río P, Navarro S, Bueren JA. Río P, et al. Hum Gene Ther. 2018 Oct;29(10):1114-1123. doi: 10.1089/hum.2018.124. Hum Gene Ther. 2018. PMID: 30117331 Review.
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: rio p. 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.
454 results