Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I
新生儿基因疗法可有效预防小鼠I型粘多糖贮积症模型中的疾病表现。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2025.101544
De Ponti, Giada; Santi, Ludovica; Dina, Giorgia; Pievani, Alice; Donsante, Samantha; Riminucci, Mara; Corsi, Alessandro; Khan, Shaukat; Passerini, Laura; Annoni, Andrea; Gregori, Silvia; Crippa, Stefania; Biondi, Andrea; Quattrini, Angelo; Tomatsu, Shunji; Aiuti, Alessandro; Bernardo, Maria Ester; Serafini, Marta