An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II.
Das S, Rruga F, Montepeloso A, Dimartino A, Spadini S, Corre G, Patel J, Cavalca E, Ferro F, Gatti A, Milazzo R, Galy A, Politi LS, Rizzardi GP, Vallanti G, Poletti V, Biffi A.
Das S, et al. Among authors: milazzo r.
Mol Ther. 2024 Mar 6;32(3):619-636. doi: 10.1016/j.ymthe.2024.01.034. Epub 2024 Feb 3.
Mol Ther. 2024.
PMID: 38310355