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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Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts by Virginia Nieto-Romero, Aida García-Torralba, Andrea Molinos-Vicente, Francisco José Moya, Sandra Rodríguez-Perales, Ramón García-Escudero, Eduardo Salido, José-Carlos Segovia, María García-Bravo

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Upregulation of NKG2D ligands impairs hematopoietic stem cell function in Fanconi anemia by José A. Casado, Antonio Valeri, Rebeca Sanchez-Domínguez, Paula Vela, Andrea López, Susana Navarro, Omaira Alberquilla, Helmut Hanenberg, Roser Pujol, José-Carlos Segovia, Jordi Minguillón, Jordi Surrallés, Cristina Díaz de Heredia, Julián Sevilla, Paula Rio, Juan A. Bueren

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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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Worldwide study of hematopoietic allogeneic stem cell transplantation in pyruvate kinase deficiency by Stephanie van Straaten, Marc Bierings, Paola Bianchi, Kensuke Akiyoshi, Hitoshi Kanno, Isabel Badell Serra, Jing Chen, Xiaohang Huang, Eduard van Beers, Supachai Ekwattanakit, Tayfun Güngör, Wijnanda Adriana Kors, Frans Smiers, Reinier Raymakers, Lucrecia Yanez, Julian Sevilla, Wouter van Solinge, Jose Carlos Segovia, Richard van Wijk

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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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