Whole-body correction of Pompe disease by AAV liver-mediated gene transfer of engineered secretable GAA transgenes
F. Puzzo,P. Colella, M. G. Biferi,D. Bali,N. K. Paulk,P. Vidal,F. Collaud, M. Simon-Sola, S. Charles,R. Hardet,C. Leborgne, A. Meliani,M. Cohen-Tannoudji,S. Astord,B. Gjata, P. Sellier,L. van Wittenberghe,A. Vignaud,F. Boisgerault,M. Barkats, P. Laforet,M. A. Kay,D. D. Koeberl,G. Ronzitti, F. Mingozzi HUMAN GENE THERAPY(2017)
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