Liver expression of secretable GAA rescues advanced Pompe disease at the biochemical, functional, and transcriptional level in Gaa mice
Umut Cagin(Inserm), Federico Mingozzi(Spark Therapeutics (United States)), Catalina Abad(Normandie Université), Nathalie Danièle(Genethon (France)), Laetitia van Wittenberghe(Genethon (France)), Giuseppe Ronzitti(Inserm), Bernard Gjata(Inserm), Pauline Sellier(Inserm), Pasqualina Colella(Stanford Medicine), Nicolas Guerchet(Inserm), Francesco Puzzo(Inserm), Manuel J. Gómez(Spanish National Centre for Cardiovascular Research), Jacomine Krijnse‐Locker(Paul Ehrlich Institut), Maryse Moya‐Nilges(Institut Pasteur)
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