Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
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M. Kay | P. Laforêt | G. Ronzitti | D. Bali | Laetitia van Wittenberghe | C. Leborgne | D. Koeberl | A. Vignaud | F. Mingozzi | N. Paulk | B. Gjata | S. Charles | F. Collaud | M. Barkats | R. Hardet | F. Boisgérault | S. Astord | F. Puzzo | P. Sellier | P. Colella | M. Biferi | M. Cohen-Tannoudji | M. Simon-Sola | Amine Meliani | P. Vidal | L. van Wittenberghe