Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
肝脏表达分泌型酸性 α-葡萄糖苷酶对小鼠晚期庞贝病的救治
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2020.05.025
Umut Cagin, Francesco Puzzo, Manuel Jose Gomez, Maryse Moya-Nilges, Pauline Sellier, Catalina Abad, Laetitia Van Wittenberghe, Nathalie Daniele, Nicolas Guerchet, Bernard Gjata, Fanny Collaud, Severine Charles, Marcelo Simon Sola, Olivier Boyer, Jacomina Krijnse-Locker, Giuseppe Ronzitti, Pasqualina