Prion Disease Therapeutics

朊病毒病治疗

• 批准号: 8745518
• 负责人: BYRON CAUGHEY
• 金额: $ 13.12万
• 依托单位: NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/8745518
• 关键词: Amyloid; Animals; Bovine Spongiform Encephalopathy; Brazil; Characteristics; Chronic Wasting Disease; Creutzfeldt-Jakob Syndrome; Disease; Extramural Activities; Goals; Human; Libraries; Neurodegenerative Disorders; Pathogenesis; PrPSc Proteins; Prion Diseases; Prions; Research Personnel; Scrapie; Sheep; System; Testing; Therapeutic; Therapeutic Agents; amyloid formation; cervid; design; novel; novel therapeutics; protein misfolding; therapeutic target

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.

可传播的海绵状脑病（TSE或PRION疾病）是致命的不可治疗的神经退行性疾病，例如Crapie，Creutzfeldt-Jakob病（CJD），牛spongine Spongiorment spongiorment Chiormanty和慢性浪费疾病（CWD）。 TSE发病机理涉及被感染宿主中称为PRPRE的异常错误折叠蛋白的积累。在2013财年：1）我们已经与外壁外研究人员合作，以识别设计的TRPZIP-3β-发行蛋白抑制两个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型化合物图书馆中寻找新的反植物化合物。已经确定了许多新化合物，它们可以阻止PRP形成并具有其他有吸引力的特征，但是需要对这些化合物进行进一步的测试，以更好地确定其治疗潜力。