Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
Umut Cagin(Inserm), Federico Mingozzi(Spark Therapeutics (United States)), Maryse Moya‐Nilges(Institut Pasteur), Catalina Abad(Normandie Université), Nathalie Danièle(Genethon (France)), Laetitia van Wittenberghe(Genethon (France)), Olivier Boyer(Inserm), Fanny Collaud(Inserm), Giuseppe Ronzitti(Inserm), Bernard Gjata(Inserm), Pauline Sellier(Inserm), Pasqualina Colella(Stanford Medicine), Nicolas Guerchet(Inserm), Marcelo Simon Sola(Inserm), Francesco Puzzo(Inserm), Manuel J. Gómez(Spanish National Centre for Cardiovascular Research), Jacomina Krijnse‐Locker(Institut Pasteur), Séverine Charles(Inserm)
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