Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
Francesco Puzzo(Inserm), Federico Mingozzi(Spark Therapeutics (United States)), Nicole K. Paulk(University of California, San Francisco), Mathilde Cohen-Tannoudji(Inserm), Laetitia van Wittenberghe(Genethon (France)), Alban Vignaud(Genethon (France)), Mark A. Kay(Io Therapeutics (United States)), Pascal Laforêt(Assistance Publique – Hôpitaux de Paris), Stéphanie Astord(Inserm), Romain Hardet(Inserm), Fanny Collaud(Inserm), Dwight D. Koeberl(Duke Medical Center), Giuseppe Ronzitti(Inserm), Bernard Gjata(Inserm), Christian Leborgne(Inserm), Maria Grazia Biferi(Inserm), Pauline Sellier(Inserm), Deeksha Bali(MRC Epidemiology Unit), Marcelo Simon‐Sola(Inserm), Pasqualina Colella(Stanford Medicine), Patrice Vidal(Inserm), Florence Boisgérault(Inserm), Amine Meliani(Inserm), Martine Barkats(Inserm), Séverine Charles(Inserm)
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