Giulia Consiglieri, Francesca Tucci, Maurizio De Pellegrin, Barbara Guerrini, Alessandro Cattoni, Giulia Risca, Stefano Scarparo, Marina Sarzana, Silvia Pontesilli, Renata Mellone, Serena Gasperini, Stefania Galimberti, Paolo Silvani, Chiara Filisetti, Silvia Darin, Giulia Forni, Simona Miglietta, Ludovica Santi, Marcella Facchini, Ambra Corti, Francesca Fumagalli, Maria Pia Cicalese, Valeria Calbi, Maddalena Migliavacca, Federica Barzaghi, Francesca Ferrua, Vera Gallo, Salvatore Recupero, Daniele Canarutto, Matteo Doglio, Lucia Tedesco, Nicola Volpi, Attilio Rovelli, Giancarlo la Marca, Maria Grazia Valsecchi, Stefano Zancan, Fabio Ciceri, Luigi Naldini, Cristina Baldoli, Rossella Parini, Bernhard Gentner, Alessandro Aiuti, Maria Ester Bernardo
Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic hematopoietic stem cell transplantation (HSCT). Autologous hematopoietic stem progenitor cell-gene therapy (HSPC-GT) provides superior metabolic correction in patients with MPSIH compared with HSCT; however, its ability to affect skeletal manifestations is unknown. Eight patients with MPSIH (mean age at treatment: 1.9 years) received lentiviral-based HSPC-GT in a phase 1/2 clinical trial (NCT03488394)...
May 2024: Science Translational Medicine