Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice
Fañanas-Baquero, Sara, Orman, Israel, Becerra Aparicio, Federico, Bermudez de Miguel, Silvia, Garcia Merino, Jordi, Yañez, Rosa, Fernandez Sainz, Yolanda, Sánchez, Rebeca, Dessy-Rodríguez, Mercedes, Alberquilla, Omaira, Alfaro, David, Zapata, Agustin, Bueren, Juan A, Segovia, Jose Carlos, Quintana-Bustamante, Oscar
Published in Haematologica (Roma) (01.06.2021)
Published in Haematologica (Roma) (01.06.2021)
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Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
Casado, Jose A, Valeri, Antonio, Sanchez-Domínguez, Rebeca, Vela, Paula, Lopez, Andrea, Navarro, Susana, Alberquilla, Omaira, Hanenberg, Helmut, Pujol, Roser, Segovia, Jose C, Minguillón, Jordi, Surrallés, Jordi, Diaz-de-Heredia, Cristina, Sevilla, Julián, Rio, Paula, Bueren, Juan A
Published in The Journal of clinical investigation (01.08.2022)
Published in The Journal of clinical investigation (01.08.2022)
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Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis
Fernandez-Perez, Raquel, Lopez-Santalla, Mercedes, Sánchez-Domínguez, Rebeca, Alberquilla, Omaira, Gutiérrez-Cañas, Irene, Juarranz, Yasmina, Bueren, Juan A, Garin, Marina I
Published in Frontiers in immunology (28.06.2021)
Published in Frontiers in immunology (28.06.2021)
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Toward Tumor Fight and Tumor Microenvironment Remodeling: PBA Induces Cell Cycle Arrest and Reduces Tumor Hybrid Cells' Pluripotency in Bladder Cancer
Rubio, Carolina, Avendaño-Ortiz, José, Ruiz-Palomares, Raquel, Karaivanova, Viktoriya, Alberquilla, Omaira, Sánchez-Domínguez, Rebeca, Casalvilla-Dueñas, José Carlos, Montalbán-Hernández, Karla, Lodewijk, Iris, Rodríguez-Izquierdo, Marta, Munera-Maravilla, Ester, Nunes, Sandra P, Suárez-Cabrera, Cristian, Pérez-Crespo, Miriam, Martínez, Víctor G, Morales, Lucía, Pérez-Escavy, Mercedes, Alonso-Sánchez, Miguel, Lozano-Rodríguez, Roberto, Cueto, Francisco J, Aguirre, Luis A, Guerrero-Ramos, Félix, Paramio, Jesús M, López-Collazo, Eduardo, Dueñas, Marta
Published in Cancers (07.01.2022)
Published in Cancers (07.01.2022)
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Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Fañanas-Baquero, Sara, Quintana-Bustamante, Oscar, Dever, Daniel P., Alberquilla, Omaira, Sanchez-Dominguez, Rebeca, Camarena, Joab, Ojeda-Perez, Isabel, Dessy-Rodriguez, Mercedes, Turk, Rolf, Schubert, Mollie S., Lattanzi, Annalisa, Xu, Liwen, Lopez-Lorenzo, Jose L., Bianchi, Paola, Bueren, Juan A., Behlke, Mark A., Porteus, Matthew, Segovia, Jose-Carlos
Published in Molecular therapy. Methods & clinical development (10.09.2021)
Published in Molecular therapy. Methods & clinical development (10.09.2021)
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The downregulated membrane expression of CD18 in CD34 + cells defines a primitive population of human hematopoietic stem cells
Mesa-Núñez, Cristina, Leon-Rico, Diego, Aldea, Montserrat, Damián, Carlos, Sanchez-Baltasar, Raquel, Sanchez, Rebeca, Alberquilla, Omaira, Segovia, José Carlos, Bueren, Juan Antonio, Almarza, Elena
Published in Stem cell research & therapy (28.04.2020)
Published in Stem cell research & therapy (28.04.2020)
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Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes
Sevilla, Julián, Navarro, Susana, Rio, Paula, Sánchez-Domínguez, Rebeca, Zubicaray, Josune, Gálvez, Eva, Merino, Eva, Sebastián, Elena, Azqueta, Carmen, Casado, José A., Segovia, José C., Alberquilla, Omaira, Bogliolo, Massimo, Román-Rodríguez, Francisco J., Giménez, Yari, Larcher, Lise, Salgado, Rocío, Pujol, Roser M., Hladun, Raquel, Castillo, Ana, Soulier, Jean, Querol, Sergi, Fernández, Jesús, Schwartz, Jonathan, García de Andoín, Nagore, López, Ricardo, Catalá, Albert, Surralles, Jordi, Díaz-de-Heredia, Cristina, Bueren, Juan A.
Published in Molecular therapy. Methods & clinical development (10.09.2021)
Published in Molecular therapy. Methods & clinical development (10.09.2021)
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In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9
López-Manzaneda, Sergio, Ojeda-Pérez, Isabel, Zabaleta, Nerea, García-Torralba, Aída, Alberquilla, Omaira, Torres, Raúl, Sánchez-Domínguez, Rebeca, Torella, Laura, Olivier, Emmanuel, Mountford, Joanne, Ramírez, Juan C., Bueren, Juan A., González-Aseguinolaza, Gloria, Segovia, Jose-Carlos
Published in Molecular therapy. Methods & clinical development (11.12.2020)
Published in Molecular therapy. Methods & clinical development (11.12.2020)
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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment
Navarro, Susana, Quintana-Bustamante, Oscar, Sanchez-Dominguez, Rebeca, Lopez-Manzaneda, Sergio, Ojeda-Perez, Isabel, Garcia-Torralba, Aida, Alberquilla, Omaira, Law, Kenneth, Beard, Brian C., Bastone, Antonella, Rothe, Michael, Villanueva, Mariela, Ramirez, Juan C., Fañanas-Baquero, Sara, Nieto-Romero, Virginia, Molinos-Vicente, Andrea, Gutierrez, Sonia, Nicoletti, Eileen, García-Bravo, María, Bueren, Juan A., Schwartz, Jonathan D., Segovia, Jose-Carlos
Published in Molecular therapy. Methods & clinical development (10.09.2021)
Published in Molecular therapy. Methods & clinical development (10.09.2021)
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Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells
Garate, Zita, Quintana-Bustamante, Oscar, Crane, Ana M., Olivier, Emmanuel, Poirot, Laurent, Galetto, Roman, Kosinski, Penelope, Hill, Collin, Kung, Charles, Agirre, Xabi, Orman, Israel, Cerrato, Laura, Alberquilla, Omaira, Rodriguez-Fornes, Fatima, Fusaki, Noemi, Garcia-Sanchez, Felix, Maia, Tabita M., Ribeiro, Maria L., Sevilla, Julian, Prosper, Felipe, Jin, Shengfang, Mountford, Joanne, Guenechea, Guillermo, Gouble, Agnes, Bueren, Juan A., Davis, Brian R., Segovia, Jose C.
Published in Stem cell reports (08.12.2015)
Published in Stem cell reports (08.12.2015)
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Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
Río, Paula, Navarro, Susana, Wang, Wei, Sánchez-Domínguez, Rebeca, Pujol, Roser M, Segovia, José C, Bogliolo, Massimo, Merino, Eva, Wu, Ning, Salgado, Rocío, Lamana, María L, Yañez, Rosa M, Casado, José A, Giménez, Yari, Román-Rodríguez, Francisco J, Álvarez, Lara, Alberquilla, Omaira, Raimbault, Anna, Guenechea, Guillermo, Lozano, M Luz, Cerrato, Laura, Hernando, Miriam, Gálvez, Eva, Hladun, Raquel, Giralt, Irina, Barquinero, Jordi, Galy, Anne, García de Andoín, Nagore, López, Ricardo, Catalá, Albert, Schwartz, Jonathan D, Surrallés, Jordi, Soulier, Jean, Schmidt, Manfred, Díaz de Heredia, Cristina, Sevilla, Julián, Bueren, Juan A
Published in Nature medicine (01.09.2019)
Published in Nature medicine (01.09.2019)
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Journal Article
Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia
Casado, Jose A, Valeri, Antonio, Sanchez-Domfnguez, Rebeca, Vela, Paula, Lopez, Andrea, Navarro, Susana, Alberquilla, Omaira, Hanenberg, Helmut, Pujol, Roser, Segovia, Jose-Carlos, Minguillon, Jordi, Surralles, Jordi, de Heredia, Cristina Diaz, Sevilla, Julian, Rio, Paula, Bueren, Juan A
Published in The Journal of clinical investigation (01.08.2022)
Published in The Journal of clinical investigation (01.08.2022)
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Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells
Fañanas-Baquero, Sara, Quintana Bustamante, Oscar, Dever, Daniel P., Alberquilla, Omaira, Sanchez-Dominguez, Rebeca, Camarena, Joab, Turk, Rolf, López Lorenzo, José Luis, Bianchi, Paola, Behlke, Mark, Bueren, Juan A, Porteus, Matt, Segovia, José C
Published in Blood (05.11.2020)
Published in Blood (05.11.2020)
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Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency
Quintana Bustamante, Oscar, Fañanas-Baquero, Sara, Dever, Daniel P., Omaira, Alberquilla, Camarena, Joab, Sanchez-Dominguez, Rebeca, Morin, Matias, Fernandez, Val, Moreno-Pelayo, Miguel Angel, Bueren, Juan A., Porteus, Matthew, Segovia, Jose C
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
Rio, Paula, Navarro, Susana, Sanchez-Dominguez, Rebeca, Segovia, Jose C, Wang, Wei, Merino, Eva, Yañez, Rosa M, Casado, Jose Antonio, Gimenez, Yari A., Roman-Rodriguez, Francisco J, Alberquilla, Omaira, Galvez, Eva, Hladun, Raquel, Barquinero, Jordi, Galy, Anne, García de Andoín, Nagore, López, Ricardo, Catalá, Albert, Cavazzana, Marina, Lefrere, Francois, Leblanc, Thierry, Schwartz, Jonathan D, Pujol, Roser, Surrallés, Jordi, Soulier, Jean, Schmidt, Manfred, Díaz de Heredia, Cristina, Sevilla, Julian, Bueren, Juan A.
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Lentivirus-mediated gene therapy corrects ribosomal biogenesis and shows promise for Diamond Blackfan anemia
Giménez, Yari, Palacios, Manuel, Sánchez-Domínguez, Rebeca, Zorbas, Christiane, Peral, Jorge, Puzik, Alexander, Ugalde, Laura, Alberquilla, Omaira, Villanueva, Mariela, Río, Paula, Gálvez, Eva, Da Costa, Lydie, Strullu, Marion, Catala, Albert, Ruiz-Llobet, Anna, Segovia, Jose Carlos, Sevilla, Julián, Strahm, Brigitte, Niemeyer, Charlotte M, Beléndez, Cristina, Leblanc, Thierry, Lafontaine, Denis Lj, Bueren, Juan, Navarro, Susana
Published in JCI insight (22.05.2024)
Published in JCI insight (22.05.2024)
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Journal Article
228. Characterization of Hematopoietic Progenitors from Pyruvate Kinase Deficient (PKD) Patients and Transduction of PKD CD34+ Cells with a Therapeutic Lentiviral Vector
Navarro, Susana, Garcia-Gomez, Maria, Lopez-Manzaneda, Sergio, Sanchez-Dominguez, Rebeca, Garcia-Bravo, Maria, Alberquilla, Omaira, Mañu, Maria M., Muriel-Ramos, Manuela, Vives-Corrons, Joan Ll, Sevilla, Julian, Bueren, Juan A., Segovia, Jose C.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Journal Article
In vitro and In vivo Genetic Disease Modelling via NHEJ-precise deletions using CRISPR/Cas9
López-Manzaneda, Sergio, Ojeda-Pérez, Isabel, Zabaleta, Nerea, Garcia-Torralba, Aída, Alberquilla, Omaira, Torres, Raúl, Sanchez-Dominguez, Rebeca, Torella, Laura, Olivier, Emmanuel, Mountford, Joanne, Ramírez, Juan C, Bueren, Juan A, González-Aseguinolaza, Gloria, Jose-Carlos Segovia
Published in bioRxiv (26.03.2020)
Published in bioRxiv (26.03.2020)
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Paper
Clinically Relevant Gene Editing in Hematopoietic Stem Cells for the Treatment of Pyruvate Kinase Deficiency Hemolytic Anemia
Fañanas-Baquero, Sara, Quintana-Bustamante, Oscar, Dever, Daniel P, Alberquilla, Omaira, Sanchez, Rebeca, Camarena, Joab, Ojeda-Perez, Isabel, Dessy-Rodriguez, Mercedes, Turk, Rolf, Schubert, Mollie S, Lopez-Lorenzo, Jose L, Bianchi, Paola, Bueren, Juan A, Behlke, Mark A, Porteus, Matthew, Jose-Carlos Segovia
Published in bioRxiv (15.01.2021)
Published in bioRxiv (15.01.2021)
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