Etuvetidigene autotemcel for the treatment of Wiskott-Aldrich Syndrome
Year: 2025
Type: article
Topic: Cellular Mechanics and Interactions
Authors and affiliations:
raw_author_name: Francesca Ferrua
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Sabina Cenciarelli
raw_affiliation_strings: Department of Biomedicine and Prevention, PhD in Immunology, Molecular Medicine and Applied Biotechnology, University of Rome Tor Vergata, Rome, Italy;
Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Stefania Giannelli
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Stefania Galimberti
raw_affiliation_strings: Bicocca Bioinformatics Biostatistics and Bioimaging B4 Center, School of Medicine and Surgery, University of Milano - Bicocca, Monza, Italy;
Biostatistics and Clinical Epidemiology, Fondazione IRCCS San Gerardo dei Tintori, Monza;
raw_author_name: Shanmuganathan Chandrakasan
raw_affiliation_strings: Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, USA;
raw_author_name: Federico Fraschetta
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Carmen Caputo
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Davide Sala
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Ilaria Monti
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Federica Barzaghi
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Valeria Calbi
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Daniele Canarutto
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: Giulia Consiglieri
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Matteo Doglio
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Francesca Fumagalli
raw_affiliation_strings: Neurology Unit and Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan, Italy;
Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Vera Gallo
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Maddalena Migliavacca
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Salvatore Recupero
raw_affiliation_strings: Department of Biomedicine and Prevention, PhD in Immunology, Molecular Medicine and Applied Biotechnology, University of Rome Tor Vergata, Rome, Italy;
Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Francesca Tucci
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Alessia Orsini
raw_affiliation_strings: Immunohematology and Transfusion Medicine Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Raffaella Milani
raw_affiliation_strings: Immunohematology and Transfusion Medicine Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Mariam Datukishvili
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Simona De Gregori
raw_affiliation_strings: Department of diagnostic medicine: clinical chemical analysis laboratory; Fondazione IRCCS Policlinico San Matteo, Pavia, Italy;
raw_author_name: Eugenio Montini
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Paolo Silvani
raw_affiliation_strings: Department of Anesthesia and Critical Care, IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Matias Soncini
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Elena Tomasetto
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
raw_author_name: Koen van Rossem
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
Karos Healthcare Consulting, Vosselaar, Belgium;
raw_author_name: Laura Castagnaro
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
raw_author_name: Federica Miotto
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
raw_author_name: Stefano Zancan
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
raw_author_name: Celeste Scotti
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
raw_author_name: Sean Russell
raw_affiliation_strings: Fondazione Telethon ETS, Rome, Italy;
PrimeRA Pharma Partners, Nottingham, UK;
raw_author_name: Luigi Naldini
raw_affiliation_strings: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: Fabio Ciceri
raw_affiliation_strings: Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: Maria Ester Bernardo
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: Suhag Parikh
raw_affiliation_strings: Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University School of Medicine, Atlanta, USA;
raw_author_name: Maria Pia Cicalese
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: Alessandro Aiuti
raw_affiliation_strings: Pediatric Immunohematology Unit and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy;
San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy;
Vita-Salute San Raffaele University, Milan, Italy;
raw_author_name: the WAS gene therapy group