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蛋白质聚集与神经退行性疾病:新型疗法的结构展望

Protein aggregation and neurodegenerative disease: Structural outlook for the novel therapeutics.

作者信息

Arar Sharif, Haque Md Anzarul, Kayed Rakez

机构信息

Mitchell Center for Neurodegenerative Diseases, University of Texas Medical Branch, Galveston, Texas, USA.

Departments of Neurology, Neuroscience and Cell Biology, University of Texas Medical Branch, Galveston, Texas, USA.

出版信息

Proteins. 2023 Aug 2. doi: 10.1002/prot.26561.

Abstract

Before the controversial approval of humanized monoclonal antibody lecanemab, which binds to the soluble amyloid-β protofibrils, all the treatments available earlier, for Alzheimer's disease (AD) were symptomatic. The researchers are still struggling to find a breakthrough in AD therapeutic medicine, which is partially attributable to lack in understanding of the structural information associated with the intrinsically disordered proteins and amyloids. One of the major challenges in this area of research is to understand the structural diversity of intrinsically disordered proteins under in vitro conditions. Therefore, in this review, we have summarized the in vitro applications of biophysical methods, which are aimed to shed some light on the heterogeneity, pathogenicity, structures and mechanisms of the intrinsically disordered protein aggregates associated with proteinopathies including AD. This review will also rationalize some of the strategies in modulating disease-relevant pathogenic protein entities by small molecules using structural biology approaches and biophysical characterization. We have also highlighted tools and techniques to simulate the in vivo conditions for native and cytotoxic tau/amyloids assemblies, urge new chemical approaches to replicate tau/amyloids assemblies similar to those in vivo conditions, in addition to designing novel potential drugs.

摘要

在可结合可溶性淀粉样β原纤维的人源化单克隆抗体lecanemab获得有争议的批准之前,所有早期可用的阿尔茨海默病(AD)治疗方法都是对症治疗。研究人员仍在努力在AD治疗药物方面取得突破,部分原因是缺乏对与内在无序蛋白质和淀粉样蛋白相关的结构信息的了解。该研究领域的主要挑战之一是了解体外条件下内在无序蛋白质的结构多样性。因此,在本综述中,我们总结了生物物理方法的体外应用,旨在阐明与包括AD在内的蛋白质病相关的内在无序蛋白质聚集体的异质性、致病性、结构和机制。本综述还将通过结构生物学方法和生物物理表征,合理化一些利用小分子调节与疾病相关的致病蛋白质实体的策略。我们还强调了模拟天然和细胞毒性tau/淀粉样蛋白组装体内条件的工具和技术,除了设计新型潜在药物外,还敦促采用新的化学方法来复制类似于体内条件的tau/淀粉样蛋白组装。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/16f3/12260341/8f8e00a5955f/PROT-93-1314-g001.jpg

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