Gene Editing for Inherited Red Blood Cell Diseases

por Quintana-Bustamante, Oscar, Fañanas-Baquero, Sara, Dessy-Rodriguez, Mercedes, Ojeda-Pérez, Isabel, Segovia, Jose-Carlos
Publicado 2022

Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides

por 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
Publicado 2023

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

por Fañanas-Baquero, Sara, Orman, Israel, Aparicio, Federico Becerra, de Miguel, Silvia Bermudez, Merino, Jordi Garcia, Yañez, Rosa, Sainz, Yolanda Fernandez, Sánchez, Rebeca, Dessy-Rodríguez, Mercedes, Alberquilla, Omaira, Alfaro, David, Zapata, Agustin, Bueren, Juan A., Segovia, Jose Carlos, Quintana-Bustamante, Oscar
Publicado 2020

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

por 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
Publicado 2021

Mutations in the RACGAP1 gene cause autosomal recessive congenital dyserythropoietic anemia type III

por 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
Publicado 2022