TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes. - Colella, P.; Puzzo, F.; Biferi, M.; Bali, D.; Paulk, N.; Vidal, P.; Collaud, F.; Simon-Sola, M.; Charles, S.; Hardet, R.; Leborgne, C.; Sellier, P.; et al. (Neuromuscular Disorders, 2017)