Advances in the gene therapy of monogenic blood cell diseases von Bueren, Juan A., Quintana‐Bustamante, Oscar, Almarza, Elena, Navarro, Susana, Río, Paula, Segovia, José C., Guenechea, Guillermo

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Targeted gene therapy and cell reprogramming in Fanconi anemia von Rio, Paula, Baños, Rocio, Lombardo, Angelo, Quintana‐Bustamante, Oscar, Alvarez, Lara, Garate, Zita, Genovese, Pietro, Almarza, Elena, Valeri, Antonio, Díez, Begoña, Navarro, Susana, Torres, Yaima, Trujillo, Juan P, Murillas, Rodolfo, Segovia, Jose C, Samper, Enrique, Surralles, Jordi, Gregory, Philip D, Holmes, Michael C, Naldini, Luigi, Bueren, Juan A

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Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA von Quintana-Bustamante, Oscar, Fañanas-Baquero, Sara, Orman, Israel, Torres, Raul, Duchateau, Philippe, Poirot, Laurent, Gouble, Agnès, Bueren, Juan A, Segovia, Jose C

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Cell fusion reprogramming leads to a specific hepatic expression pattern during mouse bone marrow derived hepatocyte formation in vivo von Quintana-Bustamante, Oscar, Grueso, Esther, Garcia-Escudero, Ramon, Arza, Elvira, Alvarez-Barrientos, Alberto, Fabregat, Isabel, Garcia-Bravo, Maria, Meza, Nestor W, Segovia, Jose C

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Gene Editing for Inherited Red Blood Cell Diseases von Quintana-Bustamante, Oscar, Fañanas-Baquero, Sara, Dessy-Rodriguez, Mercedes, Ojeda-Pérez, Isabel, Segovia, Jose-Carlos

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The Current Status of Mesenchymal Stromal Cells: Controversies, Unresolved Issues and Some Promising Solutions to Improve Their Therapeutic Efficacy von García-Bernal, David, García-Arranz, Mariano, Yáñez, Rosa M, Hervás-Salcedo, Rosario, Cortés, Alfonso, Fernández-García, María, Hernando-Rodríguez, Miriam, Quintana-Bustamante, Óscar, Bueren, Juan A, García-Olmo, Damián, Moraleda, Jose M, Segovia, José C, Zapata, Agustín G

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Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice von 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

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Mutations in the RACGAP1 gene cause autosomal recessive congenital dyserythropoietic anemia type III von Hernández, Gonzalo, Romero-Cortadellas, Lídia, Ferrer-Cortès, Xènia, Venturi, Veronica, Dessy-Rodriguez, Mercedes, Olivella, Mireia, Husami, Ammar, De Soto, Concepción Pérez, Morales-Camacho, Rosario M, Villegas, Ana, González-Fernández, Fernando-Ataulfo, Morado, Marta, Kalfa, Theodosia A, Quintana-Bustamante, Oscar, Pérez-Montero, Santiago, Tornador, Cristian, Segovia, Jose-Carlos, Sánchez, Mayka

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Enhanced anti-inflammatory effects of mesenchymal stromal cells mediated by the transient ectopic expression of CXCR4 and IL10 von Hervás-Salcedo, Rosario, Fernández-García, María, Hernando-Rodríguez, Miriam, Quintana-Bustamante, Oscar, Segovia, Jose-Carlos, Alvarez-Silva, Marcio, García-Arranz, Mariano, Minguez, Pablo, Del Pozo, Victoria, de Alba, Marta Rodríguez, García-Olmo, Damián, Ayuso, Carmen, Lamana, María Luisa, Bueren, Juan A, Yañez, Rosa María

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Highly efficient genome editing of human hematopoietic stem cells via a nano-silicon-blade delivery approach von Ma, Yuan, Han, Xin, Quintana Bustamante, Oscar, Bessa de Castro, Ricardo, Zhang, Kai, Zhang, Pengchao, Li, Ying, Liu, Zongbin, Liu, Xuewu, Ferrari, Mauro, Hu, Zhongbo, Carlos Segovia, José, Qin, Lidong

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Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides von Fañanas-Baquero, Sara, Morín, Matías, Fernández, Sergio, Ojeda-Perez, Isabel, Dessy-Rodriguez, Mercedes, Giurgiu, Miruna, Bueren, Juan A, Moreno-Pelayo, Miguel Angel, Segovia, Jose Carlos, Quintana-Bustamante, Oscar

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Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency von 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

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Hematopoietic mobilization in mice increases the presence of bone marrow-derived hepatocytes via in vivo cell fusion von QUINTANA-BUSTAMANTE, Oscar, ALVAREZ-BARRIENTOS, Alberto, KOFMAN, Alexander V, FABREGAT, Isabel, BUEREN, Juan A, THEISE, Neil D, SEGOVIA, José C

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Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment von 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

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Long-term skin regeneration in xenografts from iPSC teratoma-derived human keratinocytes von García, Marta, Quintana-Bustamante, Oscar, Segovia, José C., Bueren, Juan, Martinez-Santamaría, Lucia, Guerrero-Aspizua, Sara, Escamez, María José, Del Rio, Marcela, Larcher, Fernando

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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 von 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.

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Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme von Meza, Nestor W, Alonso-Ferrero, Maria E, Navarro, Susana, Quintana-Bustamante, Oscar, Valeri, Antonio, Garcia-Gomez, Maria, Bueren, Juan A, Bautista, Jose M, Segovia, Jose C

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CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders von Ugalde, Laura, Fañanas, Sara, Torres, Raúl, Quintana-Bustamante, Oscar, Río, Paula

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Bone marrow-derived fibrocytes participate in pathogenesis of liver fibrosis von Kisseleva, Tatiana, Uchinami, Hiroshi, Feirt, Nikki, Quintana-Bustamante, Oscar, Segovia, Jose Carlos, Schwabe, Robert F., Brenner, David A.

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Lentiviral Gene Therapy for the Correction of Congenital Dyserythropoietic Anemia Type II von Dessy-Rodriguez, Mercedes, Fañanas-Baquero, Sara, Venturi, Veronica, Payan, Salvador, Tornador, Cristian, Hernández, Gonzalo, Bianchi, Paola, Sanchez, Mayka, Ramirez, Juan Carlos, Segovia, José C, Quintana Bustamante, Oscar

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