An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II
- Resource Type
- Article
- Authors
- Das, Sabyasachi; Rruga, Fatlum; Montepeloso, Annita; Dimartino, Agnese; Spadini, Silvia; Corre, Guillaume; Patel, Janki; Cavalca, Eleonora; Ferro, Francesca; Gatti, Alessandra; Milazzo, Rita; Galy, Anne; Politi, Letterio S.; Rizzardi, Gian Paolo; Vallanti, Giuliana; Poletti, Valentina; Biffi, Alessandra
- Source
- In Molecular Therapy 6 March 2024 32(3):619-636
- Subject
- Language
- ISSN
- 1525-0016