A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease
Titel:
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease
Auteur:
Sellier, P. Collaud, F. Benchekroun, Y. Krimi Jimenez, V. Leon, X. Daniele, N. Pham, Q.H. El Andari, J. VandenDriessche, T. Chuah, M.K. Grimm, D. Bosch, F. Mingozzi, F. Ronzitti, G.