TY  - JOUR
AU  - Cagin, Umut
AU  - Puzzo, Francesco
AU  - Gomez, Manuel J
AU  - Moya-Nilges, Maryse
AU  - Sellier, Pauline
AU  - Abad, Catalina
AU  - van Wittenberghe, Laetitia
AU  - Daniele, Nathalie
AU  - Guerchet, Nicolas
AU  - Gjata, Bernard
AU  - Collaud, Fanny
AU  - Charles, Severine
AU  - Sola, Marcelo Simon
AU  - Boyer, Olivier
AU  - Krijnse-Locker, Jacomina
AU  - Ronzitti, Giuseppe
AU  - Colella, Pasqualina
AU  - Mingozzi, Federico
PY  - 2020
DO  - 10.1016/j.ymthe.2020.05.025
SN  - 1525-0024
UR  - http://hdl.handle.net/20.500.12105/13176
AB  - Pompe disease is a neuromuscular disorder caused by disease-associated variants in the gene encoding for the lysosomal enzyme acid α-glucosidase (GAA), which converts lysosomal glycogen to glucose. We previously reported full rescue of Pompe disease...
LA  - eng
PB  - Cell Press
TI  - Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.
TY  - journal article
ER  -