Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia
Authors: Lasaga, M.. et al
Journal: Haematologica. 2023 Apr;
DOI: 10.3324/haematol.2022.282418

Clustered regularly interspaced short palindromic repeats/Cas9-mediated gene editing. A promising strategy in hematological disorders
Authors: Ugalde, L. et al
Journal: Cytotherapy. 2023 Mar;
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Access to gene therapy for rare diseases when commercialization is not fit for purpose.
Authors: Fox T et al
Journal: Nat Med. 2023 Feb ;
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Improved efficacy of mesenchymal stromal cells stably expressing CXCR4 and IL-10 in a xenogeneic graft versus host disease mouse model
Authors: Hervás-Salcedo, R. et al
Journal: Frontiers in Immunology 2023 Feb;
DOI: 10.3389/fimmu.2023.1062086

Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells.
Authors: Siegner SM et al
Journal: Nat Commun. 2022
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Enforced mesenchymal stem cell tissue colonization counteracts immunopathology
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Journal: NPJ Regen Med. 2022 Oct
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Assessment of mesenchymal stem/stromal cell-based therapy in K/BxN serum transfer-induced arthritis.
Authors: Lopez-Santalla M et al
Journal: Front Immunol. 2022 Oct
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Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I
Authors: Mesa-Núñez, Cristina et al
Journal: Molecular Therapy - Methods & Clinical Development. 2022 Sep
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Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
Authors: Casado, J. A. et al
Journal: J Clin Invest. 2022 Aug
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CIBERER: Spanish national network for research on rare diseases: A highly productive collaborative initiative
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Journal: Clin Genet. 2022 May
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Gene Editing for Inherited Red Blood Cell Diseases
Authors: Quintana-Bustamante, O. et al
Journal: Frontiers in Physiology. 2022 Mar
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Comparative proteomic analysis of nuclear and cytoplasmic compartments in human cardiac progenitor cells
Authors: Albericio G. et al
Journal: Sci Rep. 2022 Jan
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Translational research for bone marrow failure patients
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Journal:  Exp Hematol. 2022 Jan
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Generation of NKX2.5(GFP) Reporter Human iPSCs and Differentiation Into Functional Cardiac Fibroblasts
Authors: Lopez-Muneta, L.  et al
Journal: Front Cell Dev Biol. 2022 Jan
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Authors: Lopez-Santalla M. et al
Biomedicines. 2021 Oct
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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment
Autores: Navarro, S. et al
Revista y Año: Mol Ther Methods Clin Dev. 2021 Jul
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Mechanism of Immunoregulatory Properties of Vasoactive Intestinal Peptide in the K/BxN Mice Model of Autoimmune Arthritis
Authors: Leceta, J, et al
Frontiers in Immunology. 2021 Jul
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Mesenchymal stem/stromal cell-based therapy for the treatment of rheumatoid arthritis: An update on preclinical studies
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EBioMedicine. 2021 Jun.
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Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis
Authors: Fernandez-Perez, Raquel, et al.
Frontiers in Immunology. 2021 Jun.
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Generating New FANCA-Deficient HNSCC Cell Lines by Genomic Editing Recapitulates the Cellular Phenotypes of Fanconi Anemia
Authors: Errazquin, Ricardo, et al.
Genes. 2021 Mar.
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GSE4-loaded nanoparticles a potential therapy for lung fibrosis that enhances pneumocyte growth, reduces apoptosis and DNA damage
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Faseb Journal. 2021 Mar;35(3):e21422..
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Next-generation Sequencing in Bone Marrow Failure Syndromes and Isolated Cytopenias: Experience of the Spanish Network on Bone Marrow Failure Syndromes
Authors: Eva Gálvez, et al.
Hemasphere. 2021 Mar 9;5(4):e539.
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The Current Status of Mesenchymal Stromal Cells: Controversies, Unresolved Issues and Some Promising Solutions to Improve Their Therapeutic Efficacy
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Front. Cell Dev. Biol. 9:650664 (2021).
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Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10
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Stem Cell Res Ther 2021 Feb 12;12(1):124.
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Generation of dyskeratosis congenita-like hematopoietic stem cells through the stable inhibition of DKC1
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Stem Cell Res Ther 2021 Jan 29;12(1):92.
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Targeted gene therapy into a safe harbor site in human hematopoietic progenitor cells
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Gene Ther. 2020 Sep;27(9):435-450 
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Theranostic Viral Nanoparticles for Autoimmune Diseases.
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J Immuno Biol Volume 5:1, 2020
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Mesenchymal Stem/Stromal Cells for Rheumatoid Arthritis Treatment: An Update on Clinical Applications
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Cells. 2020 Aug 7;9(8). 
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Human Pluripotent Stem Cell-Derived Neurons Are Functionally Mature In Vitro and Integrate into the Mouse Striatum Following Transplantation.
Authors: Comella-Bolla, A.. et al.
Mol Neurobiol. 2020 Jun;57(6):2766-2798
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Gefitinib and afatinib show potential efficacy for Fanconi anemia-related head and neck cancer
Authors: Montanuy, Helena. et al.
Clin Cancer Res 2020 Jun 15;26(12):3044-3057
DOI: 10.1158/1078-0432.ccr-19-1625

Cell Therapy With Mesenchymal Stem Cells Induces an Innate Immune Memory Response That Attenuates Experimental Colitis in the Long Term
Authors: Lopez-Santalla, M. et al.
Journal of Crohn's and Colitis, 2020, 1–12. 
DOI: 10.1093/ecco-jcc/jjaa079

Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice.
Authors: Fañanas-Baquero, S. et al.
Haematologica. 2020 Apr 30. 
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The downregulated membrane expression of CD18 in CD34(+) cells defines a primitive population of human hematopoietic stem cells
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Stem Cell Res Ther. 2020 Apr 28;11(1):164. 
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TALEN mediated gene editing in a mouse model of Fanconi anemia
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Sci Rep. 2020 Apr 24;10(1):6997
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Optimised molecular genetic diagnostics of Fanconi anaemia by whole exome sequencing and functional studies.
Authors: Bogliolo, M. et al.
Bogliolo M, et al. J Med Genet 2019;0:1–11.
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Helios modulates the maturation of a CA1 neuronal subpopulation required for spatial memory formation.
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Exp Neurol. 2020 Jan;323:113095.
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Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA

Authors: Quintana-Bustamante, O. et al.

NHEJ-Mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia

Authors: Francisco Jose Román-Rodrıguez, Laura Ugalde, Lara A´ lvarez,Begoña Díez,María José Ramırez, Cristina Risueño, Marta Cortón, Massimo Bogliolo, Sara Bernal, Francesca March, Carmen Ayuso, Helmut Hanenberg, Julián Sevilla, Sandra Rodríguez-Perales, Raúl Torres-Ruíz, Jordi Surralles,

Juan Antonio Bueren and Paula Río

Cell Stem Cell 25, 1–15.
https://doi.org/10.1016/j.stem.2019.08.016

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia, Río, Navarro et al.,
Authors: Río, P., S. Navarro, W. Wang, R. Sánchez-Domínguez, R. M. Pujol, J. C. Segovia, M. Bogliolo, E. Merino, N. Wu, R. Salgado, M. L. Lamana, R. M. Yañez, J. A. Casado, Y. Giménez, F. J. Román-Rodríguez, L. Álvarez, O. Alberquilla, A. Raimbault, G. Guenechea, M. L. Lozano, L. Cerrato, M. Hernando, E. Gálvez, R. Hladun, I. Giralt, J. Barquinero, A. Galy, N. García de Andoín, R. López, A. Catalá, J. D. Schwartz, J. Surrallés, J. Soulier, M. Schmidt, C. Díaz de Heredia, J. Sevilla and J. A. Bueren (2019).
Nature Medicine 25(9): 1396-1401.

GSE4 peptide suppresses oxidative and telomere deficiencies in ataxia telangiectasia patient cells
Authors: L. Pintado-Berninches, B. Fernandez-Varas, C. Benitez-Buelga, C. Manguan-Garcia, A. Serrano-Benitez, L. Iarriccio, J. Carrillo, G. Guenechea, S. P. Egusquiaguirre, J. L. Pedraz, R. M. Hernandez, M. Igartua, E. G. Arias-Salgado, F. Cortes-Ledesma, L. Sastre and R. Perona
Cell Death Differ,Jan 22,doi:10.1038/s41418-018-0272-7 

 c-Met Signaling Is Essential for Mouse Adult Liver Progenitor Cells Expansion After Transforming Growth Factor-beta-Induced Epithelial-Mesenchymal Transition and Regulates Cell Phenotypic Switch
Authors: L. Almale, M. Garcia-Alvaro, A. Martinez-Palacian, M. Garcia-Bravo, N. Lazcanoiturburu, A. Addante, C. Roncero, J. Sanz, O. L. M. de la, P. Bragado, W. Mikulits, V. M. Factor, S. S. Thorgeirsson, J. I. Casal, J. C. Segovia, E. Rial, I. Fabregat, B. Herrera and A. Sanchez
Stem Cells,May 20,doi:10.1002/stem.3038

Premature aging and cancer development in transgenic mice lacking functional CYLD
Authors: J. P. Alameda, A. Ramirez, R. A. Garcia-Fernandez, M. Navarro, A. Page, J. Segovia, R. Sanchez, C. Suarez-Cabrera, J. M. Paramio, A. Bravo, M. J. Fernandez-Aceñero and M. L. Casanova
AGING,11,127-159,pubmed: 

Addressing the diagnostic gaps in pyruvate kinase deficiency: Consensus recommendations on the diagnosis of pyruvate kinase deficiency
Authors: P. Bianchi, E. Fermo, B. Glader, H. Kanno, A. Agarwal, W. Barcellini, S. Eber, J. D. Hoyer, D. J. Kuter, T. M. Maia, M. D. M. Manu-Pereira, T. A. Kalfa, S. Pissard, J. C. Segovia, E. van Beers, P. G. Gallagher, D. C. Rees, R. van Wijk and t. E. R. N. i. R. H. D. with the endorsement of EuroBloodNet
Am J Hematol,Jan,94,149-161,doi:10.1002/ajh.25325

 

Inhibitory Role of Growth Hormone in the Induction and Progression Phases of Collagen-Induced Arthritis
Authors: R. Villares, G. Criado, Y. Juarranz, M. Lopez-Santalla, E. M. García-Cuesta, J. M. Rodríguez-Frade, J. Leceta, P. Lucas, J. L. Pablos, C. Martínez-A, M. I. Garin, R. P. Gomariz and M. Mellado
Frontiers in Immunology, 2018 May 25,9, PMC5980961 doi:10.3389/fimmu.2018.01165

Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency.
Authors: V. Straaten, P. Bierings, P. Bianchi, K. Akiyoshi, H. Kanno, I. B. Serra, X. Chen, E. Huang, V. Beers, S. Ekwattaanakit, T. Gungor, W. A. Kors, F. Smiers, R. Raymakers, L. Yanez, J. Sevilla, V. Solinge, C. Segovia and V. Wijk
haematologica,  PMC5792292 doi:10.3324/haematol.2017.177857

Advances in Gene Therapy for Fanconi Anemia
Authors: P. Rio, S. Navarro and J. A. Bueren
Hum Gene Ther,Oct,29,1114-1123,pubmed: 30117331 doi:10.1089/hum.2018.124

Comparative Analysis between the In Vivo Biodistribution and Therapeutic Efficacy of Adipose-Derived Mesenchymal Stromal Cells Administered Intraperitoneally in Experimental Colitis
M. Lopez-Santalla, P. Mancheno-Corvo, A. Escolano, R. Menta, O. Delarosa, J. M. Redondo, J. A. Bueren, W. Dalemans, E. Lombardo and M. I. Garin
Int J Mol Sci,Jun 23,19,PMC6073850 doi:10.3390/ijms19071853

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors
Author: M. Holstein, C. Mesa-Nunez, C. Miskey, E. Almarza, V. Poletti, M. Schmeer, E. Grueso, J. C. Ordonez Flores, D. Kobelt, W. Walther, M. K. Aneja, J. Geiger, H. B. Bonig, Z. Izsvak, M. Schleef, C. Rudolph, F. Mavilio, J. A. Bueren, G. Guenechea and Z. Ivics
Mol Ther,Apr 4,26,1137-1153,pubmed: PMC6079369 doi:10.1016/j.ymthe.2018.01.012

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases
Author: E. Almarza Novoa, S. Kasbekar, A. J. Thrasher, D. B. Kohn, J. Sevilla, T. Nguyen, J. D. Schwartz and J. A. Bueren J Allergy Clin Immunol Pract,Jul - Aug,6,1418-1420 e10,pubmed: doi:10.1016/j.jaip.2017.12.008

Improved Hematopoietic Gene Therapy in a Mouse Model of Fanconi Anemia Mediated by Mesenchymal Stromal Cells.
Authors: Fernández-García M, Luisa Lamana M, Hernando-Rodríguez M, Sánchez-Domínguez R, Bueren J, Yáñez R.
Hum Gene Ther; 2018 Mar. - PubMed; PMID: 28816065 Doi: 10.1089/hum.2017.076

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency
Authors: H. H. Abdul-Razak, C. J. Rocca, S. J. Howe, M. E. Alonso-Ferrero, J. Wang, R. Gabriel, C. C. Bartholomae, C. H. V. Gan, M. I. Garín, A. Roberts, M. P. Blundell, V. Prakash, F. J. Molina-Estevez, J. Pantoglou, G. Guenechea, M. C. Holmes, P. D. Gregory, C. Kinnon, C. von Kalle, M. Schmidt, J. A. Bueren, A. J. Thrasher and R. J. Yáñez-Muñoz
Scientific Reports,8,doi:10.1038/s41598-018-26439-9

 

 

Induced Pluripotency and Gene Editing in Fanconi Anemia
Authors:  Navarro,S,Giorgetti, A, Raya, A., Tolar,J.
Curr Gene Ther; 2017 ;16(5):321-328 - PubMed; PMID: 28103772 Doi: 10.2174/1566523217666170118112050

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia.
Authors:Adair JE, Sevilla J, de Heredia CD, Becker PS, Kiem HP, Bueren J.A
Curr Gene Ther; 2017 Jan PubMed; PMID: 28103787 Doi: 10.2174/156652321766617011911 3029

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+cells from Fanconi anemia patients.
Authors: Río P, Navarro S, Guenechea G, Sánchez-Domínguez R, Lamana ML, Yáñez R, Casado JA, Mehta PA, Pujol MR, Surrallés J, Charrier S, Galy A, Segovia JC, Díaz de Heredia C, Sevilla J, Bueren JA
Blood; 2017 09. 130(13):1535-1542 PubMed; PMID: 28801449 Doi: 10.1182/blood-2017-03-774174

Measles virus envelope pseudotyped lentiviral vectors transduce quiescent human HSCs at an efficiency without precedent.
Authors: 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
Blood Adv; 2017 Oct. 1(23):2088-2104 PubMed; PMID: 29296856 Doi: 10.1182/bloodadvances.2017007773

Biodistribution and Efficacy of Human Adipose-Derived Mesenchymal Stem Cells Following Intranodal Administration in Experimental colitis.
Authors: Lopez-Santalla M, Mancheño-Corvo P, Escolano A, Menta R, DelaRosa O, Abad JL, Büscher D, Redondo JM, Bueren JA, Dalemans W, Lombardo E, Garin MI
Front Immunol; 2017 8:638;  PubMed; PMID: 28642759 Doi: 10.3389/fimmu.2017.00638 ;

Mutations in XLF/NHEJ1/Cernunnos gene results in downregulation of telomerase genes expression and telomere shortening
Authors: 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
Hum Mol Genet; 2017 05. 26(10):1900-1914; PubMed; PMID: 28369633 Doi: 10.1093/hmg/ddx098

Highly efficient genome editing of human hematopoietic stem cells via a nano-silicon-blade delivery approach
Authors: Ma Y, Han X, Quintana Bustamante O, Bessa de Castro R, Zhang K, Zhang P, Li Y, Liu Z, Liu X, Ferrari M, Hu Z, Carlos Segovia J, Qin L
Integr Biol ; 2017 Jun. 9(6):548-554; PubMed; PMID: 28513735 Doi: 10.1039/c7ib00060j

.Efficient Recreation of t(11;22) EWSR1-FLI1+in Human Stem Cells Using CRISPR/Cas9
Authors: Torres-Ruiz R, Martinez-Lage M, Martin MC, Garcia A, Bueno C, Castaño J, Ramirez JC, Menendez P, Cigudosa JC, Rodriguez-Perales S
Stem Cell Reports; 2017 May. 8(5):1408-1420;  PubMed; PMID: 28494941 Doi: 10.1016/j.stemcr.2017.04.014

BMP4 Induces M2 Macrophage Polarization and Favors Tumor Progression in Bladder Cancer.
Authors: Martínez VG, Rubio C, Martínez-Fernández M, Segovia C, López-Calderón F, Garín MI, Teijeira A, Munera-Maravilla E, Varas A, Sacedón R, Guerrero F, Villacampa F, de la Rosa F, Castellano D, López-Collazo E, Paramio JM, Vicente Á, Dueñas M
Clin Cancer Res; 2017 Dec. 23(23):7388-7399; PubMed; PMID: 28928159 Doi: 10.1158/1078-0432.CCR-17-1004

Detectable clonal mosaicism in blood as a biomarker of cancer risk in Fanconi anemia
Authors:Reina-Castillón J, Pujol R, López-Sánchez M, Rodríguez-Santiago B, Aza-Carmona M, González JR, Casado JA, Bueren JA, Sevilla J, Badel I, Català A, Beléndez C, Dasí MÁ, Díaz de Heredia C, Soulier J, Schindler D, Pérez-Jurado LA, Surrallés J
Blood Adv; 2017 Jan. 1(5):319-329;PubMed; PMID: 29296947 Doi: 10.1182/bloodadvances.2016000943

Intralymphatic Administration of Adipose Mesenchymal Stem Cells Reduces the Severity of Collagen-Induced Experimental Arthritis
Authors: Mancheño-Corvo P, Lopez-Santalla M, Menta R, DelaRosa O, Mulero F, Del Rio B, Ramírez C, Büscher D, Bueren JA, Lopez-Belmonte J, Dalemans W, Garin MI, Lombardo E
Immunol; 2017 8:462; PubMed; PMID: 28484460 Doi: 10.3389/fimmu.2017.00462

.Gene Therapy in Fanconi anemia: a matter of time, safety and gene transfer tool efficiency.
Authors: Verhoeyen E, Rodríguez FJ, Cosset FL, Lévy C, Rio P
Curr Gene Ther; 2017 01. PubMed; PMID: 28067165 Doi: 10.2174/1566523217666170

 .A Short and Efficient Transduction Protocol for Mouse Hematopoietic Stem Cells with Lentiviral Vectors.
Authors:Fernández-García M, Mesa C, Almarza E, Bueren J, Yáñez R
Hum Gene Ther Methods; 2017 Dec. 28(6):310-317;  PubMed; PMID: 28817977 Doi: 10.1089/hgtb.2017.100 

In vivo imaging of lung inflammation with neutrophil-specific68Ga nano-radiotracer.
Authors: Pellico J, Lechuga-Vieco AV, Almarza E, Hidalgo A, Mesa-Nuñez C, Fernández-Barahona I, Quintana JA, Bueren J, Enríquez JA, Ruiz-Cabello J, Herranz F
Sci Rep; 2017 Oct. 7(1):13242; - PubMed; PMID: 29038592 Doi: 10.1038/s41598-017-12829-y 

IKKB-Mediated Resistance to Skin Cancer Development IsInk4a/Arf-Dependent.
Authors: Page A, Bravo A, Suarez-Cabrera C, Alameda JP, Casanova ML, Lorz C, Segrelles C, Segovia JC, Paramio JM, Navarro M, Ramirez A
Mol Cancer Res; 2017 Sep. 15(9):1255-1264; PubMed; PMID: 28584022 Doi: 10.1158/1541-7786.MCR-17-0157

Therapeutic gene editing in CD34+hematopoietic progenitors from Fanconi anemia patients.
Authors: 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
EMBO Mol Med; 2017 Nov. 9(11):1574-1588; PubMed; PMID: 28899930 Doi: 10.15252/emmm.201707540

 

 

 

 

 

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I.
Authors: Leon-Rico D, Aldea M, Sanchez-Baltasar R, Mesa-Nuñez C, Record J, Burns SO, Santilli G, Thrasher AJ, Bueren JA, Almarza E
Hum Gene Ther. 2016 May; Doi: 10.1089/hum.2016.016 PubMed; PMID: 27056660 

Safe and Efficient Preclinical Gene Therapy for Pyruvate Kinase Deficiency.
Authors: Garcia-Gomez M, Calabria A, Garcia-Bravo M, Benedicenti F, Kosinski P, López-Manzaneda S, Hill C, Mañu-Pereira MD, Martín MA, Orman I, Kung C, Schambach A, Jin S, Bueren JA, Montini E, Navarro S, Segovia JC
Mol Ther; 2016 May Doi: 10.1038/mt.2016.87 PubMed; PMID: 27138040

Terapia génica en línea germinal: Aspectos científicos y éticos

Authors: Bueren, J and D. Gracia
Ética en la investigación de las enfermedades raras; Capítulo de libro; 2016 (147-162)

Long-term skin regeneration in xenografts from iPSC teratoma-derived human keratinocytes.

Authors: García M, Quintana-Bustamante O, Segovia JC, Bueren J, Martinez-Santamaría L, Guerrero-Aspizua S, Escamez MJ, Del Rio M, Larcher F
Exp Dermatol 2016 Apr. Doi: 10.1111/exd.13049 PubMed; PMID: 27095005

Reprogramming human B cells into induced pluripotent stem cells and its enhancement by C/EBPa.
Authors: Bueno C, Sardina JL, Di Stefano B, Romero-Moya D, Muñoz-López A, Ariza L, Chillón MC, Balanzategui A, Castaño J, Herreros A, Fraga MF, Fernández A, Granada I, Quintana-Bustamante O, Segovia JC, Nishimura K, Ohtaka M, Nakanishi M, Graf T, Menendez P
Leukemia 2016 Mar; 30(3):674-82 Doi: 10.1038/leu.2015.294 PubMed; PMID: 26500142

Dissecting the role of epidermal growth factor receptor catalytic activity during liver regeneration and hepatocarcinogenesis.
Authors: López-Luque J, Caballero-Díaz D, Martinez-Palacián A, Roncero C, Moreno-Càceres J, García-Bravo M, Grueso E, Fernández A, Crosas-Molist E, García-Álvaro M, Addante A, Bertran E, Valverde AM, González-Rodríguez Á, Herrera B, Montoliu L, Serrano T, Segovia JC, Fernández M, Ramos E, Sánchez A, Fabregat I
Hepatology. 2016 Feb; 63(2):604-19 Doi: 10.1002/hep.28134 PubMed; PMID: 26313466

Survival and biodistribution of xenogenic adipose mesenchymal stem cells is not affected by the degree of inflammation in arthritis.
Authors: Toupet K, Maumus M, Luz-Crawford P, Lombardo E, Lopez-Belmonte J, van Lent P, Garin MI, van den Berg W, Dalemans W, Jorgensen C, Noël D
PLoS One. 2015 10(1) Doi: 10.1371/journal.pone.0114962. PubMed; PMID: 25559623

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs.
Authors: Molina-Estevez FJ, Nowrouzi A, Lozano ML, Galy A, Charrier S, von Kalle C, Guenechea G, Bueren JA, Schmidt M
Curr Gene Ther. 2015 15(6):550-62 PubMed; PMID: 26415575

Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells.
Authors: Garate Z, Quintana-Bustamante O, Crane AM, Olivier E, Poirot L, Galetto R, Kosinski P, Hill C, Kung C, Agirre X, Orman I, Cerrato L, Alberquilla O, Rodriguez-Fornes F, Fusaki N, Garcia-Sanchez F, Maia TM, Ribeiro ML, Sevilla J, Prosper F, Jin S, Mountford J, Guenechea G, Gouble A, Bueren JA, Davis BR, Segovia JC
Stem Cell Reports. 2015 Dec 8; 5(6):1053-66 Doi: 10.1016/j.stemcr.2015.10.002 PubMed; PMID: 26549847

Human Adipose-Derived Mesenchymal Stem Cells Modulate Experimental Autoimmune Arthritis by Modifying Early Adaptive T Cell Responses.
Authors: Lopez-Santalla M, Mancheño-Corvo P, Menta R, Lopez-Belmonte J, DelaRosa O, Bueren JA, Dalemans W, Lombardo E, Garin MI
Stem Cells; 2015 Dec 33(12): 3493-503 Doi: 10.1002/stem.2113 PubMed; PMID: 26205964

Epigenetic Alterations in Fanconi Anaemia: Role in Pathophysiology and Therapeutic Potential.
Authors: Lopez-Santalla M, Mancheño-Corvo P, Menta R, Lopez-Belmonte J, DelaRosa O, Bueren JA, Dalemans W, Lombardo E, Garin MI
PLoS One; 2015 Oct 14;10(10) e0139740 Doi: 10.1371/journal.pone.0139740 PubMed; PMID: 26466379

The NFKB Inducing Kinase Modulates Hematopoiesis During Stress.
Authors: González-Murillo Á, Fernández L, Baena S, Melen GJ, Sánchez R, Sánchez-Valdepeñas C, Segovia JC, Liou HC, Schmid R, Madero L, Fresno M, Ramírez M
Stem Cells; 2015 Sept 33(9) 2825-37 Doi: 10.1002/stem.2066 PubMed; PMID: 26037670

Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse transplantation model.
Authors: Fernández-García M, Yañez RM, Sánchez-Domínguez R, Hernando-Rodriguez M, Peces-Barba M, Herrera G, O'Connor JE, Segovia JC, Bueren JA, Lamana ML
Stem Cell Res Ther; 2015 Sep 7(6):165 Doi: 10.1186/s13287-015-0155-5 PubMed; PMID: 26345192

Th17 polarization of memory Th cells in early arthritis: the vasoactive intestinal peptide effect.
Authors: Jimeno R, Leceta J, Garín M, Ortiz AM, Mellado M, Rodríguez-Frade JM, Martínez C, Pérez-García S, Gomariz RP, Juarranz Y
J Leukoc Biol. 2015 Aug;98(2):257-69. Doi: 10.1189/jlb.3A0714-327R. PubMed; PMID: 25957307

The NFKB Inducing Kinase (NIK) modulates hematopoiesis during stress.
Authors: González-Murillo A, Fernández L, Baena S, Melen GJ, Sánchez R, Sánchez-Valdepeñas C, Segovia JC, Liou HC, Schmid R, Madero L, Fresno M, Ramírez M.
Stem Cells. 2015 Jun;49(2):132-41. Doi: 10.1002/stem.2066. PubMed; PMID: 26037670

Expression of genes involved in immune response and in vitro immunosuppressive effect of equine MSCs.
Authors: Remacha AR, Barrachina L, Álvarez-Arguedas S, Ranera B, Romero A, Vázquez FJ, Zaragoza P, Yañez R, Martín-Burriel I, Rodellar C
Vet Immunol Immunopathol. 2015 Jun; 165(3-4):107-18. Doi: 10.1016/j.vetimm.2015.04.004. PubMed; PMID: 25977164

Interacting resident epicardium-derived fibroblasts and recruited bone marrow cells form myocardial infarction scar.
Authors: Ruiz-Villalba A, Simón AM, Pogontke C, Castillo MI, Abizanda G, Pelacho B, Sánchez-Domínguez R, Segovia JC, Prósper F, Pérez-Pomares JM
J Am Coll Cardiol. 2015 May; 65(19): 2057-66. Doi: 10.1016/j.jacc.2015.03.520. PubMed; PMID: 25975467

The pathogenic Th profile of human activated memory Th cells in early rheumatoid arthritis can be modulated by VIP.
Authors: Jimeno R, Gomariz RP, Garín M, Gutiérrez-Cañas I, González-Álvaro I, Carrión M, Galindo M, Leceta J, Juarranz Y
J Mol Med (Berl); 2015 Apr 93(4): 457-67 Doi: 10.1007/s00109-014-1232-4  PubMed; PMID: 25430993

Comparison of haematopoietic stem cell engraftment through the retro-orbital venous sinus and the lateral vein: alternative routes for bone marrow transplantation in mice.
Authors: León-Rico D, Fernández-García M, Aldea M, Sánchez R, Peces-Barba M, Martinez-Palacio J, Yáñez RM, Almarza E.
Lab Anim. 2015 Apr;49(2):132-41. Doi: 10.1177/0023677214567915. Epub 2015 Jan 13. PubMed; PMID: 25586937

BCR-JAK2 fusion genes drives a myeloproliferative neoplasm in transplanted mice transplanted with bone marrow-transduced progenitor cells.
Authors: Cuesta-Domínguez Á, León-Rico D, Álvarez L, Díez B, Bodega-Mayor I, Baños R, Martín-Rey MÁ, Santos-Roncero M, Gaspar ML, Martín-Acosta P, Almarza E, Bueren JA, Río P, Fernández-Ruiz E.
J Pathol. 2015 Jun;236(2):219-28. Doi: 10.1002/path.4513. PubMed; PMID: 25664618

Parallel multifunctionalization of nanoparticles: a one-step modular approach for in vivo imaging.
Authors: Groult H, Ruiz-Cabello J, Pellico J, Lechuga-Vieco AV, Bhavesh R, Zamai M, Almarza E, Martín-Padura I, Cantelar E, Martínez-Alcázar MP, Herranz F
Bioconjug Chem. 2015 Jan;21 26(1):153-60. Doi: 10.1021/bc500536y.PubMed; PMID: 25494619 

Generation of patient-specific induced pluripotent stem cell from peripheral blood mononuclear cells by sendai reprogramming vectors.
Authors: Quintana-Bustamante, O, Segovia JC.
Methods Mol Biol. 2014 Dec 19 PubMed; PMID: 25523810

The pathogenic Th profile of human activated memory Th cells in early rheumatoid arthritis can be modulated by VIP.
Authors: Jimeno R, Gomariz RP, Garín M, Gutiérrez-Cañas I, González-Álvaro I, Carrión M, Galindo M, Leceta J, Juarranz Y. J Mol Med (Berl). 2014 Nov 18;93(4):457-67 Doi: 10.1007/s00109-014-1232-4 . Epub 2014 Nov 28. PubMed; PMID: 25430993

Vasoactive intestinal Peptide maintains the nonpathogenic profile of human th17-polarized cells.
Authors: Jimeno R, Leceta J, Martínez C, Gutiérrez-Cañas I, Carrión M, Pérez-García S, Garín M, Mellado M, Gomariz RP, Juarranz Y.J Mol Neurosci. 2014 Nov;54(3):512-25. Doi: 10.1007/s12031-014-0318-3. Epub 2014 May 8. PubMed; PMID: 24805298

Conversion of human fibroblasts into monocyte-like progenitor cells.
Authors: Pulecio J, Nivet E, Sancho-Martinez I, Vitaloni M, Guenechea G, Xia Y, Kurian L, Dubova I, Bueren J, Laricchia-Robbio L, Izpisua Belmonte JC.
Stem Cells. 2014 Nov;32(11):2923-38. Doi: 10.1002/stem.1800. PubMed; PMID: 25175072

Reduced expression of CD18 leads to the in vivo expansion of hematopoietic stem cells in mouse bone marrow.
Authors: Leon-Rico D, Aldea M, Sanchez R, Segovia JC, Weiss LA, Hidalgo A, Bueren JA, Almarza E.
Stem Cells. 2014 Oct;32(10):2794-8 Doi: 10.1002/stem.1762. PubMed; PMID: 24906078

p21 suppresses inflammation and tumorigenesis on pRB-deficient stratified epithelia.
Authors: Saiz-Ladera C, Lara MF, Garín M, Ruiz S, Santos M, Lorz C, García-Escudero R, Martínez-Fernández M, Bravo A, Fernández-Capetillo O, Segrelles C, Paramio JM.
Oncogene. 2014 Sep 11;33(37):4599-612. Doi: 10.1038/onc.2013.417. Epub 2013 Oct 14 PubMed; PMID: 24121270

Modeling Fanconi Anemia pathogenesis and therapeutics using integration-free patient iPSCs
Authors: Liu GH, Suzuki K, Li M, Qu J, Montserrat N, Tarantino C, Gu Y, Yi F, Xu X, Zhang W, Ruiz S, Plongthongkum N, Zhang K, Masuda S, Nivet E,Tsunekawa Y, Soligalla RD, Goebl A, Aizawa E, Young N, Kim Y, Dubova I, Li Y, Ren R, Benner C, del Sol A, Bueren J, Trujillo JP, Surralles J, Cappelli E, Dufour C, Rodriguez C and Izpisua-Belmonte JC.
Nature Communications. 2014 Jul 7;5:433 Doi: 10.1038/ncomms5330 PubMed; PMID: 24999918

Akt signaling leads to stem cell activation and promotes tumor development in epidermis.
Authors: Segrelles C1, García-Escudero R, Garín MI, Aranda JF, Hernández P, Ariza JM, Santos M, Paramio JM, Lorz C.
Stem Cells. 2014 Jul;32(7):1917-28. Doi: 10.1002/stem.1669. PubMed; PMID: 24504902

Targeted Gene Therapy and Cell Reprogramming in Fanconi Anemia.
Authors: Rio P, Baños R, Lobardo A. Quintana-Bustamente O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, Samper E, Surralles J, Gregory PD, Holmes MC, Naldini L, Bueren JA.
EMBO Mol Med. 2014 May 23;6(6):835-48 Doi: 10.15252/emmm.201303374 PubMed; PMID: 24859981

Development of a web course on gene therapy by the international consortium of gene therapy
Authors: Tremblay JP, Aartsma-Rus A, Bogdanove A, Ferreira MB, Bueren J, Huard J.
Mol Ther; 2014 Mar; 22;(3):482 Doi: 10.1038/mt.2014.11 PubMed; PMID: 24584076

Generation of iPSCs from genetically corrected Brca2 hypomorphic cells: implications in cell reprogramming and stem cell therapy
Authors: Navarro S, Moleiro V, Molina-Estevez FJ, Lozano ML, Chinchon R, Almarza E, Quintana-Bustamante O, Mostoslavsky G, Maetzig T, Galla M, Heinz N, Schiedlmeier B, Torres Y, Modlich U, Samper E, Río P, Segovia JC, Raya A, Güenechea G, Izpisua-Belmonte JC, Bueren JA.
Stem Cells; 2014 Feb;32(2):436-46. PubMed; PMID: 24420904 Doi: 10.1002/stem.1586.

Glycaemic control and implementation of the ADA/EASD-2006 consensus algorithm in type 2 diabetes mellitus patients in primary care in Spain.
Authors: García Soida FJ, Martínez González MM Collaborators (283)
Int J Clin Pract. 2014 Jan;68(1):28-39. Doi: 10.1111/ijcp.12251 PubMed; PMID: 24341300

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
Authors: Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, Vandendriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G
Mol Ther; (2013) 2013 Feb. 21(2):266-8
PubMed; PMID: 23369965
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Reduced efficacy of mesenchymal stromal cells in preventing graft-versus-host disease in an in vivo model of haploidentical bone marrow transplant with leukemia
Authors: Oviedo A, Yañez R, Colmenero I, Aldea M, Rubio A, Bueren JA, Lamana ML
Cell Transplant; (2013) 22(8):1381-94
PubMed; PMID: 23044223
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In comparison with palm oil, dietary nut supplementation delays the progression of atherosclerotic lesions in female apoE-deficient mice
Authors: Surra JC, Barranquero C, Torcal MP, Orman I, Segovia JC, Guillén N, Navarro MA, Arnal C, Osada J
Br J Nutr; (2013) 2013 Jan 28. 109(2):202-9
PubMed; PMID: 23302442
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Mutations in ERCC4, encoding the DNA-repair endonuclease XPF, cause Fanconi anemia
Authors: Bogliolo M, Schuster B, Stoepker C, Derkunt B, Su Y, Raams A, Trujillo JP, Minguillón J, Ramírez MJ, Pujol R, Casado JA, Baños R, Rio P, Knies K, Zúñiga S, Benítez J, Bueren JA, Jaspers NG, Schärer OD, de Winter JP, Schindler D, Surrallés J
Am J Hum Genet; (2013) 2013 May 2. 92(5):800-6
PubMed; PMID: 23623386
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Reticulocyte-prone malaria parasites predominantly invade CD71hi immature cells: implications for the development of an in vitro culture for Plasmodium vivax
Authors: Martín-Jaular L, Elizalde-Torrent A, Thomson-Luque R, Ferrer M, Segovia JC, Herreros-Aviles E, Fernández-Becerra C, Del Portillo HA.
Malar J.; 2013 Dec 1;12:434.
PubMed; PMID: 24289105
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Gene Therapy for Erythroid Metabolic Inherited Diseases
Authors: Maria Garcia-Gomez, Oscar Quintana-Bustamante, Maria Garcia-Bravo, S. Navarro, Zita Garate, Jose C. Segovia
Gene Therapy - Tools and Potential Applications. Molina F, ed. InTech, Croatia.; (2013) 511-538

New frontier in regenerative medicine: site-specific gene correction in patient-specific induced pluripotent stem cells
Authors: Garate Z, Davis BR, Quintana-Bustamante O, Segovia JC
Hum Gene Ther; (2013) 2013 Jun. 24(6):571-83
PubMed; PMID: 23675640
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Inhibitory Effects of Marine-Derived Dna-Binding Antitumor Tetrahydroisoquinolines on the Fanconi Anemia Pathway
Authors: Martínez S, Pérez L, Galmarini CM, Aracil M, Tercero JC, Gago F, Albella B, Bueren JA
Br J Pharmacol; (2013) 2013 Oct;170(4):871-82
PubMed; PMID: 23937566
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Impaired Cell Reprogramming in Non-Homologous End Joining Deficient Cells.
Authors: Molina-Estevez FJ, Lozano ML, Navarro S, Torres Y, Grabundzija I, Ivics Z, Samper E, Bueren JA, Guenechea G
Stem Cells; (2013) 31(8): 1726-1730
PubMed; PMID: 23630174
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The value of selected in vitro and in silico methods to predict acute oral toxicity in a regulatory context: results from the European Project ACuteTox
Authors: Prieto P, Kinsner-Ovaskainen A, Stanzel S, Albella B, Artursson P, Campillo N, Cecchelli R, Cerrato L, Díaz L, Di Consiglio E, Guerra A, Gombau L, Herrera G, Honegger P, Landry C, O'Connor JE, Páez JA, Quintas G, Svensson R, Turco L, Zurich MG, Zurbano MJ, Kopp-Schneider A
Toxicol In Vitro; (2013) 2013 Jun. 27(4):1357-76
PubMed; PMID: 22922246
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DNA delivery to 'ex vivo' human liver segments
Authors: Herrero MJ, Sabater L, Guenechea G, Sendra L, Montilla AI, Abargues R, Navarro V, Aliño SF
Gene Ther; (2012) 2012 May. 19(5):504-12
PubMed; PMID: 21993175
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Cell fusion reprogramming leads to a specific hepatic expression pattern during mouse bone marrow derived hepatocyte formation in vivo
Authors: Quintana-Bustamante O, Grueso E, Garcia-Escudero R, Arza E, Alvarez-Barrientos A, Fabregat I, Garcia-Bravo M, Meza NW, Segovia JC
PLoS One; (2012) 7(3):e33945
PubMed; PMID: 22457803
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Transforming and tumorigenic activity of JAK2 by fusion to BCR: molecular mechanisms of action of a novel BCR-JAK2 tyrosine-kinase
Authors: Cuesta-Domínguez Á, Ortega M, Ormazábal C, Santos-Roncero M, Galán-Díez M, Steegmann JL, Figuera Á, Arranz E, Vizmanos JL, Bueren JA, Río P, Fernández-Ruiz E
PLoS One; (2012) 7(2):e32451
PubMed; PMID: 22384256
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Functional characterisation of the WW minimal domain for delivering therapeutic proteins by adenovirus dodecahedron
Authors: Villegas-Méndez A, Fender P, Garin MI, Rothe R, Liguori L, Marques B, Lenormand JL
PLoS One; (2012) 7(9):e45416
PubMed; PMID: 23028993
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Down-regulated expression of hsa-miR-181c in Fanconi anemia patients: implications in TNF? regulation and proliferation of hematopoietic progenitor cells
Authors: Río P, Agirre X, Garate L, Baños R, Álvarez L, San José-Enériz E, Badell I, Casado JA, Garín M, Prósper F, Bueren JA
Blood; (2012) 2012 Mar 29. 119(13):3042-9
PubMed; PMID: 22310912
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A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice
Authors: Frecha C, Costa C, Nègre D, Amirache F, Trono D, Rio P, Bueren J, Cosset FL, Verhoeyen E
Blood; (2012) 2012 Feb 2. 119(5):1139-50
PubMed; PMID: 22117040
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Unraveling the role of FANCD2 in chronic myeloid leukemia
Authors: Valeri A, Río P, Agirre X, Prosper F, Bueren JA
Leukemia; (2012) 2012 Jun. 26(6):1447-8
PubMed; PMID: 22307177
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Gene therapy for Fanconi anemia: one step closer to the clinic
Authors: Tolar J, Becker PS, Clapp DW, Hanenberg H, de Heredia CD, Kiem HP, Navarro S, Qasba P, Rio P, Schmidt M, Sevilla J, Verhoeyen E, Thrasher AJ, Bueren J
Hum Gene Ther; (2012) 2012 Feb. 23(2):141-4
PubMed (Meeting abstracts); PMID: 22248350
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Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells
Authors: Mukherjee S, Santilli G, Blundell MP, Navarro S, Bueren JA, Thrasher AJ
PLoS One; (2011) 6(3):e17565
PubMed; PMID: 21408614
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Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells
Authors: Santilli G, Almarza E, Brendel C, Choi U, Beilin C, Blundell MP, Haria S, Parsley KL, Kinnon C, Malech HL, Bueren JA, Grez M, Thrasher AJ
Mol Ther; (2011) 2011 Jan. 19(1):122-32
PubMed; PMID: 20978475
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From the Molecular Biology to the Gene Therapy of a DNA Repair Syndrome: Fanconi Anemia
Authors: Rio, P. Navarro, S. Bueren, J.
DNA Repair and Human Health; (2011) 349-372

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting
Authors: Tolar J, Adair JE, Antoniou M, Bartholomae CC, Becker PS, Blazar BR, Bueren J, Carroll T, Cavazzana-Calvo M, Clapp DW, Dalgleish R, Galy A, Gaspar HB, Hanenberg H, Von Kalle C, Kiem HP, Lindeman D, Naldini L, Navarro S, Renella R, Rio P, Sevilla J, Schmidt M, Verhoeyen E, Wagner JE, Williams DA, Thrasher AJ
Mol Ther; (2011) 2011 Jul. 19(7):1193-8
PubMed (Review); PMID: 21540837
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Exploring the link between MORF4L1 and risk of breast cancer
Authors: Martrat G, Maxwell CA, Tominaga E, Porta M, Bonifaci N, Gomez-Baldo L, Bogliolo M, Lazaro C, Blanco I, Brunet J, Aguilar H, Fernandez-Rodriguez J, Seal S, Renwick A, Rahman N, Kuhl J, Neveling K, Schindler D, Ramirez MJ, Castella M, Hernandez G, Embrace ES, Easton DF, Peock S, Cook M, Oliver CT, Frost D, Platte R, Evans DG, Lalloo F, Eeles R, Izatt L, Chu C, Davidson R, Ong KR, Cook J, Douglas F, Hodgson S, Brewer C, Morrison PJ, Porteous M, Peterlongo P, Manoukian S, Peissel B, Zaffaroni D, Roversi G, Barile M, Viel A, Pasini B, Ottini L, Putignano AL, Savarese A, Bernard L, Radice P, Healey S, Spurdle A, Chen X, Beesley J, Kconfab KC, Rookus MA, Verhoef S, Tilanus-Linthorst MA, Vreeswijk MP, Asperen CJ, Bodmer D, Ausems MG, van Os TA, Blok MJ, Meijers-Heijboer HE, Hogervorst FB, Hebon HB, Goldgar DE, Buys S, John EM, Miron A, Southey M, Daly MB, Bcfr BC, Swe-Brca SB, Harbst K, Borg A, Rantala J, Barbany-Bustinza G, Ehrencrona H, Stenmark-Askmalm M, Kaufman B, Laitman Y, Milgrom R, Friedman E, Domchek SM, Nathanson KL, Rebbeck TR, Johannsson OT, Couch FJ, Wang X, Fredericksen Z, Cuadras D, Moreno V, Pientka FK, Depping R, Caldes T, Osorio A, Benitez J, Bueren J, Heikkinen T, Nevanlinna H, Hamann U, Torres D, Caligo MA, Godwin AK, Imyanitov EN, Janavicius R, Gemo GG, Sinilnikova OM, Stoppa-Lyonnet D, Mazoyer S, Verny-Pierre C, Castera L, de Pauw A, Bignon YJ, Uhrhammer N, Peyrat JP, Vennin P, Fert Ferrer S, Collonge-Rame MA, Mortemousque I, McGuffog L, Chenevix-Trench G, Pereira-Smith OM, Antoniou AC, Ceron J, Tominaga K, Surralles J, Pujana MA.
Breast Cancer Res; 2011 Apr 5;13(2):R40. Doi: 10.1186/bcr2862 PubMed; PMID: 21466675

Fanconi anaemia: from a monogenic disease to sporadic cancer
Authors: Valeri A, Martínez S, Casado JA, Bueren JA.
Clin Transl Oncol; 2011 Apr;13(4):215-21. doi: 10.1007/s12094-011-0645-6 PubMed; PMID: 21493181

Origin, functional role, and clinical impact of Fanconi anemia FANCA mutations
Authors: Castella M, Pujol R, Callén E, Trujillo JP, Casado JA, Gille H, Lach FP, Auerbach AD, Schindler D, Benítez J, Porto B, Ferro T, Muñoz A, Sevilla J, Madero L, Cela E, Beléndez C, de Heredia CD, Olivé T, de Toledo JS, Badell I, Torrent M, Estella J, Dasí A, Rodríguez-Villa A, Gómez P, Barbot J, Tapia M, Molinés A, Figuera A, Bueren JA, Surrallés J
Blood; (2011) 2011 Apr 7. 117(14):3759-69 Doi: 10.1182/blood-2010-08-299917
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Chromosome fragility in patients with Fanconi anaemia: diagnostic implications and clinical impact
Authors: Castella M, Pujol R, Callén E, Ramírez MJ, Casado JA, Talavera M, Ferro T, Muñoz A, Sevilla J, Madero L, Cela E, Beléndez C, de Heredia CD, Olivé T, de Toledo JS, Badell I, Estella J, Dasí Á, Rodríguez-Villa A, Gómez P, Tapia M, Molinés A, Figuera Á, Bueren JA, Surrallés J
J Med Genet; (2011) 2011 Apr. 48(4):242-50 Doi: 10.1136/jmg.2010.084210
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Peritoneal repairing cells: a type of bone marrow derived progenitor cells involved in mesothelial regeneration.
Authors: Carmona R, Cano E, Grueso E, Ruiz-Villalba A, Bera TK, Gaztambide J, Segovia JC, Muñoz-Chápuli R
J Cell Mol Med. 2011 May;15(5):1200-9. Doi: 10.1111/j.1582-4934.2010.01087.x. Epub 2010 May 14.
PubMed; PMID: 20477904

Correction of SCID-X1 using an enhancerless Vav promoter
Authors: Almarza E, Zhang F, Santilli G, Blundell MP, Howe SJ, Thornhill SI, Bueren JA, Thrasher AJ
Hum Gene Ther; (2011) 2011 Mar. 22(3):263-70 Doi: 10.1089/hum.2010.119
PubMed; PMID: 20887212

Immunoresponse against the transgene limits hematopoietic engraftment of mice transplanted in utero with virally transduced fetal liver.
Authors: Alonso-Ferrero ME, Valeri A, Yañez R, Navarro S, Garin MI, Ramirez JC, Bueren JA, Segovia JC.
Gene Ther; (2011) 2011 May;18(5):469-78 Doi: 10.1038/gt.2010.160
PubMed; PMID: 21179174

Development of an in vitro model for the simultaneous study of the efficacy and hematotoxicity of antileukemic compounds
Authors: Valeri A, Alonso-Ferrero ME, Cerrato L, Martínez S, Bueren JA, Albella B
Toxicol Lett; (2010) 2010 Dec 15. 199(3):317-22 Doi: 10.1016/j.toxlet.2010.09.014
PubMed; PMID: 20883753

Targeted Lentiviral Gene Therapy to Rescue the X-CGD Phenotype
Authors: Santilli, G; Brendel, C; Almarza, E; Beilin, C; Blundell, M; Haria, S; Kinnon, C; Bueren, JA; Grez, M; Thrasher, AJ
Hum Gene Ther; (2010) 21 (4) 515-515, APR 2010

Bcr/Abl interferes with the Fanconi anemia/BRCA pathway: implications in the chromosomal instability of chronic myeloid leukemia cells
Authors: Valeri A, Alonso-Ferrero ME, Río P, Pujol MR, Casado JA, Pérez L, Jacome A, Agirre X, Calasanz MJ, Hanenberg H, Surrallés J, Prosper F, Albella B, Bueren JA
PLoS One; 2010 Dec 28; 5(12):e15525 Doi: 10.1371/journal.pone.0015525 PubMed; PMID: 21203397

Development of lentiviral vectors with optimized transcriptional activity for the gene therapy of patients with Fanconi anemia
Authors: González-Murillo A, Lozano ML, Alvarez L, Jacome A, Almarza E, Navarro S, Segovia JC, Hanenberg H, Guenechea G, Bueren JA, Río P
Hum Gene Ther; (2010) 2010 May. 21(5):623-30 Doi: 10.1089/hum.2009.141
PubMed; PMID: 20001454

Prostaglandin E2 plays a key role in the immunosuppressive properties of adipose and bone marrow tissue-derived mesenchymal stromal cells
Authors: Yañez R, Oviedo A, Aldea M, Bueren JA, Lamana ML
Exp Cell Res; (2010) 2010 Nov 15. 316(19):3109-23 Doi: 10.1016/j.yexcr.2010.08.008
PubMed; PMID: 20804749

Mesenchymal stem cells: biological properties and clinical applications
Authors: García-Gómez I, Elvira G, Zapata AG, Lamana ML, Ramírez M, Castro JG, Arranz MG, Vicente A, Bueren J, García-Olmo D.
Expert Opin Biol Ther; (2010) 10(10):1453-68 Doi: 10.1517/14712598.2010.519333
PubMed; PMID: 20831449

In vivo delivery of antigens by adenovirus dodecahedron induces cellular and humoral immune responses to elicit antitumor immunity
Authors: Villegas-Mendez A, Garin MI, Pineda-Molina E, Veratti E, Bueren JA, Fender P, Lenormand JL.
Mol Ther; 2010 May;18(5):1046-53. Doi: 10.1038/mt.2010.16. Epub 2010 Feb 23. PubMed; PMID: 20179681

A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells
Authors: Raya A, Rodriguez-Piza I, Navarro S, Richaud-Patin Y, Guenechea G, Sanchez-Danes A, Consiglio A, Bueren J, Belmonte JCI
Nat Protoc; (2010) 5 (4): 647-660; PMID: 20224565
Doi: 10.1038/nprot.2010.9

Altered hematopoiesis in mice lacking DNA polymerase mu is due to inefficient double-strand break repair
Authors: Lucas D, Escudero B, Ligos JM, Segovia JC, Estrada JC, Terrados G, Blanco L, Samper E, Bernad A.
PLoS Genet; 2009 Feb;5(2):e1000389. Doi: 10.1371/journal.pgen.1000389. PubMed; PMID: 19229323

Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients
Authors: Jacome A, Navarro S, Rio P, Yanez RM, Gonzalez-Murillo A, Lozano ML, Lamana ML, Sevilla J, Olive T, Diaz-Heredia C, Badell I, Estella J, Madero L, Guenechea G, Casado J, Segovia JC, Bueren JA
Mol Ther; (2009) 17(6):1083-1092 Doi: 10.1038/mt.2009.26
PubMed; PMID: 19277017

p73 plays a role in erythroid differentiation through GATA1 induction
Authors: Marqués-García F, Ferrandiz N, Fernández-Alonso R, González-Cano L, Herreros-Villanueva M, Rosa-Garrido M, Fernández-García B, Vaque JP, Marqués MM, Alonso ME, Segovia JC, León J, Marín MC.
J Biol Chem; (2009) 284(32):21139-56
PubMed; PMID: 19509292
Doi: 10.1074/jbc.M109.026849

In vitro sensitivity of granulo-monocytic progenitors as a new toxicological cell system and endpoint in the ACuteTox Project
Authors: Cerrato L, Valeri A, Bueren JA, Albella B
Toxicol Appl Pharmacol; (2009) 238(2):111-119 Doi: 10.1016/j.taap.2009.05.005
PubMed; PMID: 19442680

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors
Authors: Modlich U, Navarro S, Zychlinski D, Maetzig T, Knoess S, Brugman MH, Schambach A, Charrier S, Galy A, Thrasher AJ, Bueren J, Baum C
Mol Ther; (2009) 17(11): 1919-1928, NOV 2009 Doi: 10.1038/mt.2009.179
PubMed; PMID: 19672245

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells
Authors: Raya A, Rodriguez-Piza I, Guenechea G, Vassena R, Navarro S, Barrero MJ, Consiglio A, Castella M, Rio P, Sleep E, Gonzalez F, Tiscornia G, Garreta E, Aasen T, Veiga A, Verma IM, Surralles J, Bueren J, Belmonte JCI
Nature; (2009) 460(7251):53-9
PubMed; PMID: 19483674
Doi: 10.1038/nature08129

Rescue of pyruvate kinase deficiency in mice by gene therapy using the human isoenzyme.
Authors: Meza NW, Alonso-Ferrero ME, Navarro S, Quintana-Bustamante O, Valeri A, Garcia-Gomez M, Bueren JA, Bautista JM, Segovia JC.
Mol Ther; (2009) 17(12):2000-9
PubMed; PMID: 19755962
Doi: 10.1038/mt.2009.200

Bone Marrow-Derived Cells Promote Liver Regeneration in Mice With Erythropoietic Protoporphyria
Authors: Garcia-Bravo M, Moran-Jimenez MJ, Quintana-Bustamante O, Mendez M, Gutierrez-Vera I, Bueren J, Salido E, Segovia JC, Fontanellas A, de Salamanca RE
Transplantation; (2009) 88 (12): 1332-1340, Dec 27 2009
PubMed; PMID: 20029329
Doi: 10.1097/TP.0b013e3181bce00e

Increased Intraocular Insulin-like Growth Factor-I Triggers Blood-Retinal Barrier Breakdown
Authors: Haurigot V, Villacampa P, Ribera A, Llombart C, Bosch A, Nacher V, Ramos D, Ayuso E, Segovia JC, Bueren JA, Ruberte J, Bosch F
J Biol Chem; (2009) 284 (34): 22961-22969, Aug 21 2009 - PMID: 19473988
Doi: 10.1074/jbc.M109.014787

Elevated levels of IL-1beta in Fanconi anaemia group A patients due to a constitutively active phosphoinositide 3-kinase-Akt pathway are capable of promoting tumour cell proliferation.
Authors: Ibáñez A, Río P, Casado JA, Bueren JA, Fernández-Luna JL, Pipaón C.
Biochem J; (2009) 422(1):161-70
PubMed; PMID: 19473116
Doi: 10.1042/BJ20082118

Immunomagnetic enrichment of human and mouse hematopoietic stem cells for gene therapy applications.
Authors: Guenechea G, Segovia JC, Bueren JA.
Methods Mol Biol; (2009) 506:1-11
PubMed; PMID: 19110615
Doi: 10.1007/978-1-59745-409-4_1

 

IGF-I mediates regeneration of endocrine pancreas by increasing beta cell replication through cell cycle protein modulation in mice
Authors: Agudo J, Ayuso E, Jimenez V, Salavert A, Casellas A, Tafuro S, Haurigot V, Ruberte J, Segovia JC, Bueren J, Bosch F
Diabetología; (2008) 51(10): 1862-1872
PubMed; PMID: 18663428
Doi: 10.1007/s00125-008-1087-8

Evolution to pathogenicity of the parvovirus minute virus of mice in immunodeficient mice involves genetic heterogeneity at the capsid domain that determines tropism
Authors: Lopez-Bueno A, Segovia JC, Bueren JA, O'Sullivan MG, Wang F, Tattersall P, Almendral JM
J Virol; (2008) 82(3): 1195-1203
PubMed; PMID: 18045943
Doi: 10.1128/JVI.01692-07

Constitutively active Akt induces ectodermal defects and impaired bone morphogenetic protein signaling
Authors: Segrelles C, Moral M, Lorz C, Santos M, Lu J, Cascallana JL, Lara MF, Carbajal S, Martínez-Cruz AB, García-Escudero R, Beltran L, Segovia JC, Bravo A, DiGiovanni J, Paramio JM.
Mol Biol Cell; (2008) 19(1):137-49
PubMed; PMID: 17959825

Squalene in a sex-dependent manner modulates atherosclerotic lesion which correlates with hepatic fat content in apoE-knockout male mice
Authors: Guillén N, Acín S, Navarro MA, Perona JS, Arbonés-Mainar JM, Arnal C, Sarría AJ, Surra JC, Carnicer R, Orman I, Segovia JC, Ruiz-Gutiérrez V, Osada J.
Atherosclerosis; (2008) 197(1):72-83
PubMed; PMID: 17854812
Doi: 10.1016/j.atherosclerosis.2007.08.008

Fanconi Anemia Core Complex Moves to Chromatin
Authors: Rio P, Bueren JA
Blood; (2008) 111(10): 4837-4838

In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1
Authors: Rio P, Meza NW, Gonzalez-Murillo A, Navarro S, Alvarez L, Surralles J, Castella M, Guenechea G, Segovia JC, Hanenberg H, Bueren JA
Blood; (2008) 112(13): 4853-4861
PubMed; PMID: 18812474
Doi: 10.1182/blood-2008-05-156356

Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors
Authors: Gonzalez-Murillo A, Lozano ML, Montini E, Bueren JA, Guenechea G
Blood; (2008) 112(8): 3138-3147
PubMed; PMID: 18684860
Doi: 10.1182/blood-2008-03-142661

Relevance of the Fanconi anemia pathway in the response of human cells to trabectedin
Authors: Casado JA, Rio P, Marco E, Garcia-Hernandez V, Domingo A, Perez L, Tercero JC, Vaquero JJ, Albella B, Gago F, Bueren JA
Mol Cancer Ther; (2008) 7(5): 1309-1318
PubMed; PMID: 18483318
Doi: 10.1158/1535-7163.MCT-07-2432

 

Characteristics of lentiviral vectors harboring the proximal promoter of the vav proto-oncogene: A weak and efficient promoter for gene therapy
Authors: Almarza E, Rio P, Meza NW, Aldea M, Agirre X, Guenechea G, Segovia JC, Bueren JA
Mol Ther; (2007) 15(8): 1487-1494
PubMed; PMID: 17534266
Doi: 10.1038/sj.mt.6300213

Introducción a los efectos deterministas. Síndrome de médula ósea. Terapia celular y genética.
Authors: Guenechea G
Avances en Radiobiología, Serie Ponencias Editorial CIEMAT; (2007) 117-152
Book chapter

Predictive Tests of hematotoxicity in Pharmacological Cancer Therapy
Authors: Albella B, Gomez S, Guenechea G, Bueren JA
Stem Cells and Cancer; (2007) 117-141
Book chapter

Functional Knock-Down of Human RAD51 for Testing the Fanconi Anemia BRCA Connection
Authors: Rio P, Hanenberg H
Hum Genet; (2007) (15): 211-225
Book chapter

Terapia génica con células madre
Authors: Bueren JA, Guenechea G, Segovia JC
RAN d Medicina, ed Fundamentos Moleculares de la Medicina II; (2007) 165-175;
Book chapter

Aplicaciones de la terapia celular y génica para el control de la EICH
Authors: Lamana M, Yanez R, Briones J, Garcia-Castro J, Ramirez M, Sierra J, Bueren JA
Methods and Findings; (2007) (29): 23-26;
Book chapter

Olive oil preparation determines the atherosclerotic protection in apolipoprotein E knockout mice.
Authors: Acín S, Navarro MA, Perona JS, Arbonés-Mainar JM, Surra JC, Guzmán MA, Carnicer R, Arnal C, Orman I, Segovia JC, Osada J, Ruiz-Gutiérrez V.
J Nutr Biochem; (2007) 18(6):418-24
PubMed; PMID: 17049830
Doi: 10.1016/j.jnutbio.2006.08.005

A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network
Authors: Casado JA, Callen E, Jacome A, Rio P, Castella M, Lobitz S, Ferro T, Munoz A, Sevilla J, Cantalejo A, Cela E, Cervera J, Sanchez-Calero J, Badell I, Estella J, Dasi A, Olive T, Ortega JJ, Rodriguez-Villa A, Tapia M, Molines A, Madero
J Med Genet; (2007) 44(4): 241-249
PubMed; PMID: 17105750
Doi: 10.1136/jmg.2006.044719

Hypomorphic mutations in the gene encoding a key Fanconi anemia protein, FANCD2, sustain a significant group of FA-D2 patients with severe phenotype
Authors: Kalb R, Neveling K, Hoehn H, Schneider H, Linka Y, Batish SD, Hunt C, Berwick M, Callen E, Surralles J, Casado JA, Bueren J, Dasi A, Soulier J, Gluckman E, Zwaan CM, van Spaendonk R, Pals G, de Winter JP, Joenje H, Grompe M, Auerbach
Am J Hum Genet; (2007) 80(5): 895-910
PubMed; PMID: 17436244
Doi: 10.1086/517616

In Vitro and in vivo expression of human erythrocyte pyruvate kinase in erythroid cells: A gene therapy approach
Authors: Meza NW, Quintana-Bustamante O, Puyet A, Rio P, Navarro S, Diez A, Bueren JA, Bautista JM, Segovia JC
Hum Gene Ther; (2007) 18(6): 502-514
PubMed; PMID: 17547515
Doi: 10.1089/hum.2006.052