论文信息 - Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice - 字舞流文

Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

G. Ronzitti | F. Mingozzi | F. Collaud | C. Georger | D. Gross | E. Gicquel | N. Daniele | P. Sellier | P. Vidal | A. Miranda | I. Richard | L. van Wittenberghe | B. Bertin | Bertil-Froidevaux