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

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Natural estrogens enhance the engraftment of human hematopoietic stem and progenitor cells in immunodeficient mice by Sara Fañanas-Baquero, Israel Orman, Federico Becerra Aparicio, Silvia Bermudez de Miguel, Jordi Garcia Merino, Rosa Yañez, Yolanda Fernandez Sainz, Rebeca Sánchez, Mercedes Dessy-Rodríguez, Omaira Alberquilla, David Alfaro, Agustin Zapata, Juan A. Bueren, Jose Carlos Segovia, Oscar Quintana-Bustamante

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

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

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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 by Zita Garate, Oscar Quintana-Bustamante, Ana M. Crane, Emmanuel Olivier, Laurent Poirot, Roman Galetto, Penelope Kosinski, Collin Hill, Charles Kung, Xabi Agirre, Israel Orman, Laura Cerrato, Omaira Alberquilla, Fatima Rodriguez-Fornes, Noemi Fusaki, Felix Garcia-Sanchez, Tabita M. Maia, Maria L. Ribeiro, Julian Sevilla, Felipe Prosper, Shengfang Jin, Joanne Mountford, Guillermo Guenechea, Agnes Gouble, Juan A. Bueren, Brian R. Davis, Jose C. Segovia

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