Insights from Therapeutic Studies for PrP Prion Disease.
Insights from Therapeutic Studies for PrP Prion Disease.
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PrP 朊病毒病治疗研究的见解。
DOI：
10.1101/cshperspect.a024430
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发表时间：
2017
期刊：
影响因子：
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通讯作者：
Doh-ura K.
中科院分区：
文献类型：
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作者：
Teruya K;Doh-ura K.
虽然朊病毒病的有效治疗尚未建立，但在了解其发病机制方面已取得了许多进展，这促进了对该疾病治疗方法的研究。几种化合物，包括氟吡汀、奎纳克林、戊聚糖多硫酸酯和多西环素，最近已在试验基础上用于朊病毒病患者。同时，发现了几种抗朊病毒先导化合物，包括化合物B（compB）、IND系列和anle 138 B。然而，临床试验仍然远远没有产生显著有益的结果，动物中的先导化合物研究结果突出了新的挑战。这些努力突出了需要改进或进一步探索以实现更有效疗法的领域。在这项工作中，我们回顾了朊病毒相关的治疗研究的最新进展，并讨论了基本的科学问题，以解决有意义的医学干预朊病毒疾病。
Although an effective therapy for prion disease has not yet been established, many advances have been made toward understanding its pathogenesis, which has facilitated research into therapeutics for the disease. Several compounds, including flupirtine, quinacrine, pentosan polysulfate, and doxycycline, have recently been used on a trial basis for patients with prion disease. Concomitantly, several lead antiprion compounds, including compound B (compB), IND series, and anle138b, have been discovered. However, clinical trials are still far from yielding significantly beneficial results, and the findings of lead compound studies in animals have highlighted new challenges. These efforts have highlighted areas that need improvement or further exploration to achieve more effective therapies. In this work, we review recent advances in prion-related therapeutic research and discuss basic scientific issues to be resolved for meaningful medical intervention of prion disease.