Functional, biochemical and transcriptional rescue of advanced Pompe disease in mice with liver expression of secretable GAA
Umut Cagin(Inserm), Federico Mingozzi(Spark Therapeutics (United States)), Nathalie Danièle(Genethon (France)), Laetitia van Wittenberghe(Genethon (France)), Jérémy Rouillon(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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