Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

por Schott, Juliane W., León-Rico, Diego, Ferreira, Carolina B., Buckland, Karen F., Santilli, Giorgia, Armant, Myriam A., Schambach, Axel, Cavazza, Alessia, Thrasher, Adrian J.
Publicado 2019

The downregulated membrane expression of CD18 in CD34(+) cells defines a primitive population of human hematopoietic stem cells

por Mesa-Núñez, Cristina, Leon-Rico, Diego, Aldea, Montserrat, Damián, Carlos, Sanchez-Baltasar, Raquel, Sanchez, Rebeca, Alberquilla, Omaira, Segovia, José Carlos, Bueren, Juan Antonio, Almarza, Elena
Publicado 2020

Lentiviral Vector-Mediated Correction of a Mouse Model of Leukocyte Adhesion Deficiency Type I

por Leon-Rico, Diego, Aldea, Montserrat, Sanchez-Baltasar, Raquel, Mesa-Nuñez, Cristina, Record, Julien, Burns, Siobhan O., Santilli, Giorgia, Thrasher, Adrian J., Bueren, Juan A., Almarza, Elena
Publicado 2016

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

por Rai, Rajeev, Romito, Marianna, Rivers, Elizabeth, Turchiano, Giandomenico, Blattner, Georges, Vetharoy, Winston, Ladon, Dariusz, Andrieux, Geoffroy, Zhang, Fang, Zinicola, Marta, Leon-Rico, Diego, Santilli, Giorgia, Thrasher, Adrian J., Cavazza, Alessia
Publicado 2020

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

por Piras, Giuseppa, Montiel-Equihua, Claudia, Chan, Yee-Ka Agnes, Wantuch, Slawomir, Stuckey, Daniel, Burke, Derek, Prunty, Helen, Phadke, Rahul, Chambers, Darren, Partida-Gaytan, Armando, Leon-Rico, Diego, Panchal, Neelam, Whitmore, Kathryn, Calero, Miguel, Benedetti, Sara, Santilli, Giorgia, Thrasher, Adrian J., Gaspar, H. Bobby
Publicado 2020

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease

por Schejtman, Andrea, Vetharoy, Winston, Choi, Uimook, Rivat, Christine, Theobald, Narda, Piras, Giuseppa, Leon-Rico, Diego, Buckland, Karen, Armenteros-Monterroso, Elena, Benedetti, Sara, Ashworth, Michael T., Rothe, Michael, Schambach, Axel, Gaspar, Hubert Bobby, Kang, Elizabeth M., Malech, Harry L., Thrasher, Adrian J., Santilli, Giorgia
Publicado 2021