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

by Cristina Mesa-Núñez, Diego Leon-Rico, Montserrat Aldea, Carlos Damián, Raquel Sanchez-Baltasar, Rebeca Sanchez, Omaira Alberquilla, José Carlos Segovia, Juan Antonio Bueren, Elena Almarza
Published 2020-04-01

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

by Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher, Alessia Cavazza
Published 2020-08-01

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

by Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, Derek Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytan, Diego Leon-Rico, Neelam Panchal, Kathryn Whitmore, Miguel Calero, Sara Benedetti, Giorgia Santilli, Adrian J. Thrasher, H. Bobby Gaspar
Published 2020-09-01