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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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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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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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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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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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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Modelling Congenital Dyserythropoietic Anemia Type II through Gene Editing in Hematopoietic Stem and Progenitor Cells von Dessy-Rodriguez, Mercedes, Fañanas-Baquero, Sara, Venturi, Veronica, Payán-Pernía, Salvador, Tornador, Cristian, Hernandez, Gonzalo, Sanchez, Mayka, Segovia, José C, Quintana Bustamante, Oscar

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Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells von 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

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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 von 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

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Estrogens Enhance Human Hematopoietic Stem Cell Engraftment in a Xenogenic Transplantation Model von Fañanas-Baquero, Sara, Orman, Israel, Becerra Aparicio, Federico, Bermudez de Miguel, Silvia, Garcia Merino, Jordi, Yañez, Rosa M, Fernandez, Yolanda, Alfaro, David, Zapata, Agustin, Bueren, Juan A., Segovia, Jose C, Quintana Bustamante, Oscar

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von QUINTANA BUSTAMANTE, Oscar, PORTEUS, Mathew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von PORTEUS, Matthew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos, QUINTANA BUSTAMANTE, Óscar

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Pyruvate Kinase Deficiency (PKD) gene editing treatment method von PORTEUS, Matthew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos, QUINTANA BUSTAMANTE, Óscar

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von PORTEUS, Matthew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos, QUINTANA BUSTAMANTE, Óscar

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von PORTEUS, Matthew, FAÑANAS BAQUERO, Sara, SEGOVIA SANZ, José Carlos, QUINTANA BUSTAMANTE, Óscar

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von FAÑANAS BAQUERO Sara, SEGOVIA SANZ José Carlos, QUINTANA BUSTAMANTE Óscar, PORTEUS Matthew

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Edición génica en progenitores hematopoyéticos humanos para la inserción de una matriz terapeutica en el gen PKLR von Fañanás Baquero, Sara, Segovia Sanz, José Carlos, Quintana Bustamante, Óscar

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PYRUVATE KINASE DEFICIENCY (PKD) GENE EDITING TREATMENT METHOD von QUINTANA BUSTAMANTE, OSCAR, FANANAS BAQUERO, SARA, SEGOVIA SANZ, JOSE CARLOS, PORTEUS, MATTHEW

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피루베이트 키나아제 결핍증(PKD) 유전자 편집 치료 방법 von FANANAS BAQUERO SARA, PORTEUS MATTHEW, SEGOVIA SANZ JOSE CARLOS, QUINTANA BUSTAMANTE OSCAR

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