Cheng Biao, Gong Hao, Xiao Hongwen, Petersen Robert B, Zheng Ling, Huang Kun
Tongji School of Pharmacy, Huazhong University of Science and Technology, Wuhan, People's Republic of China.
Biochim Biophys Acta. 2013 Oct;1830(10):4860-71. doi: 10.1016/j.bbagen.2013.06.029. Epub 2013 Jun 29.
The deposition of self-assembled amyloidogenic proteins is associated with multiple diseases, including Alzheimer's disease, Parkinson's disease and type 2 diabetes mellitus. The toxic misfolding and self-assembling of amyloidogenic proteins are believed to underlie protein misfolding diseases. Novel drug candidates targeting self-assembled amyloidogenic proteins represent a potential therapeutic approach for protein misfolding diseases.
In this perspective review, we provide an overview of the recent progress in identifying inhibitors that block the aggregation of amyloidogenic proteins and the clinical applications thereof.
Compounds such as polyphenols, certain short peptides, and monomer- or oligomer-specific antibodies, can interfere with the self-assembly of amyloidogenic proteins, prevent the formation of oligomers, amyloid fibrils and the consequent cytotoxicity.
Some inhibitors have been tested in clinical trials for treating protein misfolding diseases. Inhibitors that target the aggregation of amyloidogenic proteins bring new hope to therapy for protein misfolding diseases.
自组装淀粉样蛋白的沉积与多种疾病相关，包括阿尔茨海默病、帕金森病和2型糖尿病。淀粉样蛋白的毒性错误折叠和自组装被认为是蛋白质错误折叠疾病的基础。针对自组装淀粉样蛋白的新型候选药物代表了一种治疗蛋白质错误折叠疾病的潜在方法。
在本观点性综述中，我们概述了在鉴定阻断淀粉样蛋白聚集的抑制剂及其临床应用方面的最新进展。
多酚、某些短肽以及单体或寡聚体特异性抗体等化合物可干扰淀粉样蛋白的自组装，防止寡聚体、淀粉样纤维的形成以及随之而来的细胞毒性。
一些抑制剂已在治疗蛋白质错误折叠疾病的临床试验中进行了测试。针对淀粉样蛋白聚集的抑制剂为蛋白质错误折叠疾病的治疗带来了新希望。
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