Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
利用腺相关病毒(AAV)介导的肝脏递送可分泌酸性α-葡萄糖苷酶挽救小鼠庞贝氏症
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.aam6375
Puzzo, Francesco; Colella, Pasqualina; Biferi, Maria G; Bali, Deeksha; Paulk, Nicole K; Vidal, Patrice; Collaud, Fanny; Simon-Sola, Marcelo; Charles, Severine; Hardet, Romain; Leborgne, Christian; Meliani, Amine; Cohen-Tannoudji, Mathilde; Astord, Stephanie; Gjata, Bernard; Sellier, Pauline; van Wittenberghe, Laetitia; Vignaud, Alban; Boisgerault, Florence; Barkats, Martine; Laforet, Pascal; Kay, Mark A; Koeberl, Dwight D; Ronzitti, Giuseppe; Mingozzi, Federico