Centre for Neuroscience Research, University of Edinburgh, UK.
Prion. 2009 Jul-Sep;3(3):121-8. doi: 10.4161/pri.3.3.9289. Epub 2009 Jul 16.
Insights into the molecular basis and the temporal evolution of neurotoxicity in prion disease are increasing, and recent work in mice leads to new avenues for targeting treatment of these disorders. Using lentivirally mediated RNA interference (RNAi) against native prion protein (PrP), White et al. report the first therapeutic intervention that results in neuronal rescue, prevents symptoms and increases survival in mice with established prion disease.(1) Both the target and the timing of treatment here are crucial to the effectiveness of this strategy: the formation of the neurotoxic prion agent is prevented at a point when diseased neurons can still be saved from death. But the data also give new insights into the timing of treatment in the context of the pattern of spread of prion infection throughout the brain, with implications for developing the most effective treatments.
朊病毒病的神经毒性的分子基础和时间演变的研究不断深入，最近在小鼠身上的研究为这些疾病的靶向治疗开辟了新途径。White 等人利用慢病毒介导的 RNA 干扰（RNAi）针对天然朊病毒蛋白（PrP），报告了首例可导致神经元挽救、预防症状并提高已建立的朊病毒病小鼠存活率的治疗干预措施。(1) 该策略的有效性取决于目标和治疗时机这两个关键因素：在患病神经元仍有可能免于死亡的时间点，阻止了神经毒性朊病毒制剂的形成。但这些数据也为治疗时机提供了新的见解，阐明了朊病毒感染在大脑中的传播模式，这对开发最有效的治疗方法具有重要意义。
