Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I. - De Ponti G; Santi L; Dina G; Pievani A; Donsante S; Riminucci M; Corsi A; Khan S; Passerini L; Annoni A; Gregori S; Crippa S; et al. (Molecular therapy. Methods & clinical development, 2025)