Centro de Investigación en Sanidad (CISA-INIA), Ctra de Algete a El Casar, Valdeolmos, Madrid, Spain.
J Infect Dis. 2011 Oct 1;204(7):1038-45. doi: 10.1093/infdis/jir484.
Prion diseases, which are mostly represented in humans by Creutzfeldt-Jakob disease, are transmissible neurodegenerative disorders characterized by vacuolization and neuronal loss, as well as by the accumulation of an abnormal form of the prion protein. These disorders have yet no effective treatment, and drugs that block prion replication in vitro do not significantly slow down the progression of the disease when used in vivo at late stages. Cell therapy that has been already tested in other neurodegenerative disorders therefore represents an interesting alternative approach. In this study, we showed for the first time in prion diseases that intracerebral transplantation of fetal neural stem cells significantly extended both incubation and survival time. This result was dependant on the time window chosen for the engraftment and was obtained with both genetically modified and wild-type stem cells, therefore forging a path toward efficient stem cell therapy for human prion diseases.
朊病毒病，在人类中主要表现为克雅氏病，是一种可传播的神经退行性疾病，其特征是出现空泡化和神经元丧失，以及异常形式的朊病毒蛋白积累。这些疾病目前尚无有效的治疗方法，而在体内晚期使用体外阻断朊病毒复制的药物并不能显著减缓疾病的进展。因此，已经在其他神经退行性疾病中进行测试的细胞疗法代表了一种有趣的替代方法。在这项研究中，我们首次在朊病毒病中表明，脑内移植胎儿神经干细胞显著延长了潜伏期和存活时间。该结果依赖于移植选择的时间窗口，并且使用基因修饰和野生型干细胞都获得了该结果，因此为人类朊病毒病的有效干细胞治疗开辟了道路。
