Prion Disease Therapeutics

传染性海绵状脑病（tse或朊病毒疾病）是一种致命的无法治愈的神经退行性疾病，如痒病、克雅氏病（CJD）、牛海绵状脑病和慢性消耗性疾病（CWD）。TSE的发病机制涉及一种被称为PrPres的异常错误折叠蛋白在被感染宿主体内的积累。2013财年：1)我们与校外研究人员合作，确定了一种设计好的Trpzip-3发夹，可以抑制两种不同淀粉样蛋白系统中的淀粉样蛋白形成。2)我们继续从合作者在巴西合成的新化合物文库中寻找新的抗朊病毒化合物。已经发现了许多新的化合物可以阻断PrP-res的形成，并具有其他吸引人的特征，但需要对这些化合物进行进一步的测试，以更好地确定它们的治疗潜力。

The transmissible spongiform encephalopathies (TSEs or prion diseases)are fatal untreatable neurodegenerative diseases such as scrapie, Creutzfeldt-Jakob disease (CJD), bovine spongiform encephalopathy and chronic wasting disease (CWD). TSE pathogenesis involves the accumulation of an abnormal misfolded protein, called PrPres, in infected hosts. In FY 2013: 1) We have collaborated with extramural investigators to identify a designed Trpzip-3 β-hairpin that inhibits amyloid formation in two different amyloid systems. 2) We have continued to search for new anti-prion compounds from libraries of novel compounds that have been synthesized in Brazil by our collaborators. A number of new compounds have been identified that block PrP-res formation and have other attractive characteristics, but further testing of these compounds will be required to better establish their therapeutic potential.