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目的观察携带cdc2-小干扰RNA(cdc2-siRNA)的重组腺相关病毒(rAAV)小脑注射对C型尼曼-皮克病(NPC)小鼠行为学的影响。方法2周龄npc-/-小鼠经小脑注射携带cdc2-siRNA的rAAV,动态测量小鼠体重,并进行衣架悬挂实验及足印实验评估小鼠4~8周的运动能力。结果(1)cdc2-siRNA组小鼠体重减轻明显延缓;(2)衣架悬挂实验显示cdc2-siRNA组小鼠运动缺陷明显改善;(3)足印实验显示cdc2-siRNA组相对步距[(75.1±8.6)%]与对照病毒组及非手术组相比明显增加。结论小脑注射携带cdc2-siRNA的rAAV改善了npc-/-小鼠的行为学,有可能为NPC的治疗提供新的有效途径。
Objective To observe the effects of recombinant adeno-associated virus (rAAV) carrying cdc2-siRNA on the behavior of C-type Niemann-Pick disease (NPC) mice. Methods 2 weeks old npc - / - mice were injected rAAV carrying cdc2-siRNA into the cerebellum to measure the weight of the mice dynamically. The hanger suspension test and footprinting test were performed to evaluate the 4 ~ 8 weeks’ exercise ability. (2) The hanger suspension test showed that the motor deficit in cdc2-siRNA group was significantly improved; (3) The footprinting experiments showed that the relative pace of cdc2-siRNA group [(75.1 ± 8.6%)] was significantly increased compared with the control and non-surgical groups. Conclusion Cerebellar injection of rAAV carrying cdc2-siRNA improves the behavior of npc - / - mice and may provide a new and effective approach for the treatment of NPC.