Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
Pasqualina Colella(Stanford Medicine), Federico Mingozzi(Spark Therapeutics (United States)), Maryse Moya‐Nilges(Institut Pasteur), Mathilde Cohen-Tannoudji(Inserm), Nathalie Danièle(Genethon (France)), Laetitia van Wittenberghe(Genethon (France)), Fanny Collaud(Inserm), Guillaume Tanniou(Inserm), Bernard Gjata(Inserm), Maria Grazia Biferi(Inserm), Pauline Sellier(Inserm), Marcelo Simon‐Sola(Inserm), Nicolas Guerchet(Inserm), Umut Cagin(Inserm), Manuel J. Gómez(Spanish National Centre for Cardiovascular Research), Jacomina Krijnse‐Locker(Institut Pasteur), Séverine Charles(Inserm)
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