Enhanced Susceptibility of Galectin-1 Deficient Mice to Experimental Colitis

by Raquel Fernandez-Perez, Raquel Fernandez-Perez, Mercedes Lopez-Santalla, Mercedes Lopez-Santalla, Rebeca Sánchez-Domínguez, Rebeca Sánchez-Domínguez, Omaira Alberquilla, Omaira Alberquilla, Irene Gutiérrez-Cañas, Yasmina Juarranz, Juan A. Bueren, Juan A. Bueren, Marina I. Garin, Marina I. Garin
Published 2021-06-01

In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

by Sergio López-Manzaneda, Isabel Ojeda-Pérez, Nerea Zabaleta, Aída García-Torralba, Omaira Alberquilla, Raúl Torres, Rebeca Sánchez-Domínguez, Laura Torella, Emmanuel Olivier, Joanne Mountford, Juan C. Ramírez, Juan A. Bueren, Gloria González-Aseguinolaza, Jose-Carlos Segovia
Published 2020-12-01

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

by Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P. Dever, Omaira Alberquilla, Rebeca Sanchez-Dominguez, Joab Camarena, Isabel Ojeda-Perez, Mercedes Dessy-Rodriguez, Rolf Turk, Mollie S. Schubert, Annalisa Lattanzi, Liwen Xu, Jose L. Lopez-Lorenzo, Paola Bianchi, Juan A. Bueren, Mark A. Behlke, Matthew Porteus, Jose-Carlos Segovia
Published 2021-09-01

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

by Susana Navarro, Oscar Quintana-Bustamante, Rebeca Sanchez-Dominguez, Sergio Lopez-Manzaneda, Isabel Ojeda-Perez, Aida Garcia-Torralba, Omaira Alberquilla, Kenneth Law, Brian C. Beard, Antonella Bastone, Michael Rothe, Mariela Villanueva, Juan C. Ramirez, Sara Fañanas-Baquero, Virginia Nieto-Romero, Andrea Molinos-Vicente, Sonia Gutierrez, Eileen Nicoletti, María García-Bravo, Juan A. Bueren, Jonathan D. Schwartz, Jose-Carlos Segovia
Published 2021-09-01

Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes

by Julián Sevilla, Susana Navarro, Paula Rio, Rebeca Sánchez-Domínguez, Josune Zubicaray, Eva Gálvez, Eva Merino, Elena Sebastián, Carmen Azqueta, José A. Casado, José C. Segovia, Omaira Alberquilla, Massimo Bogliolo, Francisco J. Román-Rodríguez, Yari Giménez, Lise Larcher, Rocío Salgado, Roser M. Pujol, Raquel Hladun, Ana Castillo, Jean Soulier, Sergi Querol, Jesús Fernández, Jonathan Schwartz, Nagore García de Andoín, Ricardo López, Albert Catalá, Jordi Surralles, Cristina Díaz-de-Heredia, Juan A. Bueren
Published 2021-09-01