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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-/-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-/-小鼠神经元细胞骨架损伤的保护作用。结果(1)携带cdc2-siRNA的rAAV明显减少npc-/-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SMI31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-/-小鼠神经元细胞骨架损害具有保护作用。
Objective To evaluate whether cerebellar injection of recombinant adeno-associated virus (cdA2) carrying cdc2-siRNA has protective effects on neuronal cytoskeletal damage in type C Niemann-Pick disease (NPC) mice. Methods The recombinant adeno-associated virus (cdA2) carrying cdc2-siRNA was injected into the cerebellum of 2-week-old npc - / - mice. Immunohistochemistry and Western blotting were used to observe the cytoskeleton damage of npc - / - mice The protective effect. Results (1) rAAV carrying cdc2-siRNA significantly reduced the number of npc - / - mouse neurite outgrowth; (2) rAAV carrying cdc2-siRNA effectively inhibited phosphorylated neurofilament (identified by SMI31) and phosphorylated Tau protein (recognized by PHF-1). Conclusion Cerebellar injection of rAAV carrying cdc2-siRNA has a protective effect on neuronal cytoskeletal damage in npc - / - mice.