Department of Neurology, David Geffen School of Medicine, Brain Research Institute, and Molecular Biology Institute, University of California, Los Angeles, CA, United States.
Prog Mol Biol Transl Sci. 2019;168:277-287. doi: 10.1016/bs.pmbts.2019.07.010. Epub 2019 Aug 7.
Disease-modifying therapies for proteinopathies are urgently needed yet clinical trials for the major neurodegenerative diseases, Alzheimer's and Parkinson's, have been failing at an alarming rate leaving patients and caregivers scrambling for any sign of hope. At the same time, for one family of proteinopathies, the rare TTR amyloidoses, disease-modifying therapy has existed for almost 3 decades and two new types of disease-modifying therapy have become available more recently. In this chapter, I discuss those therapies, examine to what extent they can be generalized for other diseases, and consider what we may learn from their relative success.
治疗蛋白构象病的疗法亟待开发，而针对阿尔茨海默病和帕金森病等主要神经退行性疾病的临床试验却以惊人的速度失败，使患者及其护理人员四处寻找一线希望。与此同时，对于罕见的转甲状腺素蛋白淀粉样变性病这一类蛋白构象病，治疗该病的疗法已经存在近 30 年，而最近又出现了两种新的治疗该病的疗法。在本章中，我将讨论这些疗法，研究它们在多大程度上可以推广到其他疾病，并思考我们可以从它们的相对成功中学到什么。
