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Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa mice
Cagin, Umut, Puzzo, Francesco, Gomez, Manuel Jose, Moya-Nilges, Maryse, Sellier, Pauline, Abad, Catalina, Van Wittenberghe, Laetitia, Daniele, Nathalie, Guerchet, Nicolas, Gjata, Bernard, Krijnse-LockVolume:
129
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2019.11.070
Date:
February, 2020
File:
PDF, 340 KB
2020