Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized NAGLU vector
通过向实质内或池内递送衣壳修饰的 AAV8 密码子优化的 NAGLU 载体来纠正 IIIB 型粘多糖贮积症小鼠的疾病
期刊:Human Molecular Genetics
影响因子:3.1
doi:10.1093/hmg/ddac209
Courtney J Rouse, Kimberley Hawkins, Nadia Kabbej, Justin Dalugdug, Aishwarya Kunta, Mi-Jung Kim, Shinichi Someya, Zachary Herbst, Michael Gelb, Isabella Dinelli, Elizabeth Butterworth, Darin J Falk, Erinn Rosenkrantz, Hamza Elmohd, Hamid Khaledi, Samar Mowafy, Frederick Ashby, Coy D Heldermon