Laboratory of Prion Biology, Department of Neuroscience, Scuola Internazionale Superiore Di Studi Avanzati (SISSA), Trieste, Italy.
Expert Opin Ther Pat. 2021 Dec;31(12):1097-1115. doi: 10.1080/13543776.2021.1945033. Epub 2021 Jul 21.
: Prion diseases are a class of rare and fatal neurodegenerative diseases for which no cure is currently available. They are characterized by conformational conversion of cellular prion protein (PrP) into the disease-associated 'scrapie' isoform (PrP). Under an etiological point of view, prion diseases can be divided into acquired, genetic, and idiopathic form, the latter of which are the most frequent.: Therapeutic approaches targeting prion diseases are based on the use of chemical and nature-based compounds, targeting either PrP or PrP or other putative player in pathogenic mechanism. Other proposed anti-prion treatments include passive and active immunization strategies, peptides, aptamers, and PrP-directed RNA interference techniques. The treatment efficacy has been mainly assessed in cell lines or animal models of the disease testing their ability to reduce prion accumulation.: The assessed strategies focussing on the identification of an efficient anti-prion therapy faced various issues, which go from permeation of the blood brain barrier to immunological tolerance of the host. Indeed, the use of combinatory approaches, which could boost a synergistic anti-prion effect and lower the potential side effects of single treatments and may represent an extreme powerful and feasible way to tackle prion disease.
朊病毒病是一类罕见且致命的神经退行性疾病，目前尚无治愈方法。它们的特征是细胞朊病毒蛋白（PrP）构象转换为疾病相关的“瘙痒”异构体（PrP）。从病因学角度来看，朊病毒病可分为获得性、遗传性和特发性形式，后者最为常见。针对朊病毒病的治疗方法基于使用化学和天然化合物，针对 PrP 或 PrP 或其他潜在的致病机制参与者。其他提出的抗朊病毒治疗包括被动和主动免疫策略、肽、适体和针对 PrP 的 RNA 干扰技术。治疗效果主要在疾病的细胞系或动物模型中进行评估，测试它们减少朊病毒积累的能力。评估的策略侧重于确定有效的抗朊病毒治疗方法，面临着各种问题，包括穿透血脑屏障和宿主的免疫耐受。事实上，联合使用可能会产生协同抗朊病毒作用并降低单一治疗的潜在副作用的组合方法，可能是治疗朊病毒病的一种极其强大且可行的方法。
