Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
全身性rAAV9-hNAGLU递送治疗粘多糖贮积症IIIB的可行性和安全性:灵长类动物毒理学、生物分布和免疫学评估
期刊:Human Gene Therapy Clinical Development
影响因子:
doi:10.1089/humc.2013.208
Murrey, Darren A; Naughton, Bartholomew J; Duncan, F Jason; Meadows, Aaron S; Ware, Tierra A; Campbell, Katie J; Bremer, William G; Walker, Christopher M; Goodchild, Laurie; Bolon, Brad; La Perle, Krista; Flanigan, Kevin M; McBride, Kim L; McCarty, Douglas M; Fu, Haiyan