Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

158 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
Fañanas-Baquero S, Morín M, Fernández S, Ojeda-Perez I, Dessy-Rodriguez M, Giurgiu M, Bueren JA, Moreno-Pelayo MA, Segovia JC, Quintana-Bustamante O. Fañanas-Baquero S, et al. Among authors: bueren ja. Front Genome Ed. 2023 Apr 28;5:1104666. doi: 10.3389/fgeed.2023.1104666. eCollection 2023. Front Genome Ed. 2023. PMID: 37188156 Free PMC article.
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.
Role of Hospital Exemption in Europe: position paper from the Spanish Advanced Therapy Network (TERAV).
Sánchez-Guijo F, Avendaño-Solá C, Badimón L, Bueren JA, Canals JM, Delgadillo J, Delgado J, Eguizábal C, Fernández-Santos ME, García-Olmo D, González-Aseguinolaza G, Juan M, Martín F, Mata R, Montserrat N, Pérez-Martínez A, Pérez-Simón JA, Prósper F, Urbano-Ispizua Á, Zapata AG, Sureda A, Moraleda JM. Sánchez-Guijo F, et al. Among authors: bueren ja. Bone Marrow Transplant. 2023 Jun;58(6):727-728. doi: 10.1038/s41409-023-01962-0. Epub 2023 Mar 25. Bone Marrow Transplant. 2023. PMID: 36966215 Free PMC article. No abstract available.
Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia.
Lasaga M, Río P, Vilas-Zornoza A, Planell N, Navarro S, Alignani D, Fernández-Varas B, Mouzo D, Zubicaray J, Pujol RM, Nicoletti E, Schwartz JD, Sevilla J, Ainciburi M, Ullate-Agote A, Surrallés J, Perona R, Sastre L, Prosper F, Gomez-Cabrero D, Bueren JA. Lasaga M, et al. Among authors: bueren ja. Haematologica. 2023 Oct 1;108(10):2652-2663. doi: 10.3324/haematol.2022.282418. Haematologica. 2023. PMID: 37021532 Free PMC article.
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: bueren ja. 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.
Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia.
Casado JA, Valeri A, Sanchez-Domínguez R, Vela P, López A, Navarro S, Alberquilla O, Hanenberg H, Pujol R, Segovia JC, Minguillón J, Surrallés J, de Heredia CD, Sevilla J, Rio P, Bueren JA. Casado JA, et al. Among authors: bueren ja. J Clin Invest. 2022 Aug 1;132(15):e142842. doi: 10.1172/JCI142842. J Clin Invest. 2022. PMID: 35671096 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: bueren ja. 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.
158 results