Institute for Regenerative Medicine and Biotherapies (IRMB), Neural Stem Cell, MSC and Neurodegenerative Diseases - U1183 INSERM (Institut National de la Santé et de la Recherche Médicale), 80 rue Augustin Fliche, 34295 Montpellier, France; Université de Montpellier, 163 rue Auguste Broussonet, 34090 Montpellier, France.
Institute for Regenerative Medicine and Biotherapies (IRMB), Neural Stem Cell, MSC and Neurodegenerative Diseases - U1183 INSERM (Institut National de la Santé et de la Recherche Médicale), 80 rue Augustin Fliche, 34295 Montpellier, France; Université de Montpellier, 163 rue Auguste Broussonet, 34090 Montpellier, France; Centre Hospitalo-Universitaire de Montpellier, 191 Av. du Doyen Gaston Giraud, 34295 Montpellier, France.
Curr Opin Pharmacol. 2019 Feb;44:8-14. doi: 10.1016/j.coph.2018.11.001. Epub 2018 Nov 23.
Despite multiple efforts to find treatments, prion diseases are still incurable. The currently available therapeutic strategies are mostly based on compounds to inhibit pathological PrP (PrPSc) accumulation, and cellular PrP (PrP) conversion into PrP. However, they cannot reverse the pathological changes already present in the brain. Cell-based therapeutic strategies could promote the repair of the pre-existing brain damage. The few available data come mostly from preclinical studies using neural stem cells, bone marrow-derived microglia and mesenchymal stem cells, as cell sources. Moreover, the benefits of cell-based therapeutic strategies could be linked not only to the replacement of damaged cells, but also to the secretion of trophic factors by the grafted cells that might modulate inflammation, cell death, or endogenous neurogenesis.
尽管已经进行了多次努力来寻找治疗方法，但朊病毒病仍然无法治愈。目前可用的治疗策略主要基于抑制病理性 PrP（PrPSc）积累和细胞 PrP（PrP）转化为 PrP 的化合物。然而，它们不能逆转已经存在于大脑中的病理性变化。基于细胞的治疗策略可以促进对已经存在的脑损伤的修复。少数可用的数据主要来自使用神经干细胞、骨髓来源的小胶质细胞和间充质干细胞作为细胞来源的临床前研究。此外，基于细胞的治疗策略的益处不仅可能与受损细胞的替代有关，还可能与移植细胞分泌的营养因子有关，这些因子可能调节炎症、细胞死亡或内源性神经发生。
