Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes
Pasqualina Colella,F. Puzzo, M. Biferi,Deeksha Bali,Nicole K. Paulk,Patrice Vidal,Fanny Collaud, M. Simon-Sola,Séverine Charles,Romain Hardet,Christian Leborgne, P. Sellier,L. Van Wittenberghe,Florence Boisgerault,Martine Barkats,P. Laforêt,Mark A. Kay,Dwight D. Koeberl,Giuseppe Ronzitti,Federico Mingozzi NEUROMUSCULAR DISORDERS(2017)
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