Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct
利用基于酶的脑穿梭构建体治疗由 β-葡萄糖脑苷脂酶缺乏引起的神经元溶酶体功能障碍
期刊:Nature Communications
影响因子:14.7
doi:10.1038/s41467-023-37632-4
Alexandra Gehrlein, Vinod Udayar, Nadia Anastasi, Martino L Morella, Iris Ruf, Doris Brugger, Sophia von der Mark, Ralf Thoma, Arne Rufer, Dominik Heer, Nina Pfahler, Anton Jochner, Jens Niewoehner, Luise Wolf, Matthias Fueth, Martin Ebeling, Roberto Villaseñor, Yanping Zhu, Matthew C Deen, Xiaoyang
