Prion Disease Therapeutics

传染性海绵状脑病（TSEs或朊病毒疾病）是一种致命的、无法治愈的神经退行性疾病，如瘙痒症、克雅氏病（CJD）、牛海绵状脑病和慢性消耗病（CWD）。TSE发病机制涉及在感染宿主中异常错误折叠蛋白质（称为PrPres）的积累。2013财年：1）我们与校外研究人员合作，确定了一种设计的Trpzip-3-发夹，可抑制两种不同淀粉样蛋白系统中的淀粉样蛋白形成。2)我们继续从我们的合作者在巴西合成的新化合物库中寻找新的抗朊病毒化合物。一些新的化合物已被确定，阻止PrP-res的形成，并具有其他有吸引力的特点，但这些化合物的进一步测试将需要更好地建立其治疗潜力。

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.