Centre for Misfolding Diseases, Yusuf Hamied Department of Chemistry, University of Cambridge, Cambridge, UK.
Department of Pathology, UC San Diego, San Diego, CA, USA.
Nat Struct Mol Biol. 2021 Apr;28(4):365-372. doi: 10.1038/s41594-021-00565-x. Epub 2021 Mar 25.
Prions consist of pathological aggregates of cellular prion protein and have the ability to replicate, causing neurodegenerative diseases, a phenomenon mirrored in many other diseases connected to protein aggregation, including Alzheimer's and Parkinson's diseases. However, despite their key importance in disease, the individual processes governing this formation of pathogenic aggregates, as well as their rates, have remained challenging to elucidate in vivo. Here we bring together a mathematical framework with kinetics of the accumulation of prions in mice and microfluidic measurements of aggregate size to dissect the overall aggregation reaction into its constituent processes and quantify the reaction rates in mice. Taken together, the data show that multiplication of prions in vivo is slower than in in vitro experiments, but efficient when compared with other amyloid systems, and displays scaling behavior characteristic of aggregate fragmentation. These results provide a framework for the determination of the mechanisms of disease-associated aggregation processes within living organisms.
朊病毒由细胞朊病毒蛋白的病理性聚集物组成,具有复制能力,导致神经退行性疾病,这一现象在许多与蛋白质聚集相关的其他疾病中也有体现,包括阿尔茨海默病和帕金森病。然而,尽管它们在疾病中具有重要作用,但在体内阐明导致致病性聚集物形成的各个过程及其速率仍然具有挑战性。在这里,我们将朊病毒在小鼠体内积累的动力学数学框架与微流控测量的聚集物大小相结合,将整体聚集反应分解为其组成过程,并在小鼠体内定量反应速率。总的来说,这些数据表明,朊病毒在体内的繁殖速度比体外实验慢,但与其他淀粉样蛋白系统相比效率更高,并且表现出与聚集物断裂特征相符的缩放行为。这些结果为在活生物体中确定与疾病相关的聚集过程的机制提供了一个框架。