中文摘要：

目的评估经小脑注射携带cdc2-siRNA的重组腺相关病毒（rAAV）对C型尼曼-皮克病（NPC）小鼠神经元细胞骨架损伤是否具有保护作用。方法将携带cdc2-siRNA重组腺相关病毒（rAAV）注射入2周龄npc-f-小鼠的小脑，采用免疫组织化学及免疫印迹方法观察该病毒对npc-f-小鼠神经元细胞骨架损伤的保护作用。结果（1）携带cdc2siRNA的rAAV明显减少npc-f-小鼠轴突球状体的数量；（2）携带cdc2-siRNA的rAAV有效抑制磷酸化的神经丝（由SM31识别）及磷酸化的Tau蛋白（由PHF_1识别）的表达。结论小脑注射携带cdc2-siRNA的rAAV对npc-f-小鼠神经元细胞骨架损害具有保护作用。

英文摘要：

Objective To assess the protective effect of cerebellar injection of recombinant adeno-associated virus （rAAV） encoding cde2 siRNA on the injured neuronal cytoskeleton of Niemann-Pick disease type C （NPC） mice. Methods RAAV encoding cdc2-siRNA was injected into the cerebellum of 2 weeks old npc-f- mice. The protective effect of the rAAV on the injured neuronal cytoskeleton in the treated mice was observed by immunohistology and immunoblotting technologies. Results （1）RAAV encoding cdc2-siRNA remarkably reduced the number of axonal spheroids in the treated mice. （2）rAAV encoding cdc2-siRNA markedly inhibited the expressions of phospho-neurofilament （recognized by SMI31）and phospho-Tau （recognized by PHF- 1 ）. Conclusions The rAAV encoding cdc2-siRNA injected into the cerebellum of npc-f- mice can protect the injured neuronal cytoskeleton of npc-f- mice.