对针对朊病毒疾病的有效PrP导向疗法的迫切需求。
The Compelling Demand for an Effective PrP-Directed Therapy against Prion Diseases.
作者信息
Astolfi Andrea, Spagnolli Giovanni, Biasini Emiliano, Barreca Maria Letizia
机构信息
Department of Pharmaceutical Sciences, "Department of Excellence 2018-2022", University of Perugia, 06123 Perugia PG, Italy.
Department of Cellular, Computational and Integrative Biology (CIBIO) and Dulbecco Telethon Institute, University of Trento, 38122 Trento TN, Italy.
出版信息
ACS Med Chem Lett. 2020 Nov 2;11(11):2063-2067. doi: 10.1021/acsmedchemlett.0c00528. eCollection 2020 Nov 12.
Decades of research efforts have conclusively provided overwhelming evidence that the cellular prion protein (PrP) plays a central role in prion diseases, a set of fatal and incurable neurodegenerative disorders for which no therapy is yet available. In this Viewpoint, we provide an overview of the drug discovery strategies in the field, highlighting the current therapeutic hypotheses targeting, whether directly or indirectly, PrP as well as the antiprion agents closest to clinical application.
数十年的研究工作已确凿地提供了大量证据，表明细胞朊蛋白（PrP）在朊病毒疾病中起核心作用，这是一组致命且无法治愈的神经退行性疾病，目前尚无治疗方法。在本观点文章中，我们概述了该领域的药物发现策略，重点介绍了目前直接或间接靶向PrP的治疗假说以及最接近临床应用的抗朊病毒药物。
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