Trepczyk Karolina, Er Safak, Hlushchuk Irena, Airavaara Mikko, Alwani Anna, Maziarz Katarzyna, Chmielarz Piotr, Słomska Kinga, Wieczerzak Ewa, Jankowska Elżbieta
Department of Biomedical Chemistry, Faculty of Chemistry, University of Gdansk, Wita Stwosza 63, 80-308 Gdańsk, Poland.
Pharmacology and Drug Development Division of Pharmacology and Pharmacotherapy, Faculty of Pharmacy, University of Helsinki, FI-00014 Helsinki, Finland.
J Med Chem. 2025 Apr 24;68(8):8967-8979. doi: 10.1021/acs.jmedchem.5c00645. Epub 2025 Apr 7.
The development of age-related neurodegenerative diseases is associated with the accumulation of damaged and misfolded proteins. Such proteins are eliminated from cells by proteolytic systems, mainly by 20S proteasomes, whose activity declines with age. Its stimulation has been recognized as a promising approach to delay the onset or ameliorate the symptoms of neurodegenerative disorders. Here we present peptidomimetics that are very effective in stimulating the proteasome in biochemical assays and in cell culture. They are stable in human plasma and capable of penetrating the cell membranes. The activators demonstrated the ability to enhance h20S degradation of α-synuclein and tau, whose aggregates are involved in the development of Parkinson's and Alzheimer's diseases, respectively. The peptidomimetics did not show cytotoxicity to HEK293T and primary hippocampal cells. Additionally, these compounds were highly effective in reducing the amount of phosphorylated α-synuclein aggregates in hippocampal neurons in a mouse embryonic cell model.
与年龄相关的神经退行性疾病的发展与受损和错误折叠蛋白质的积累有关。此类蛋白质通过蛋白水解系统从细胞中清除,主要是通过20S蛋白酶体,其活性会随着年龄的增长而下降。对其进行刺激已被认为是一种有前景的方法,可延缓神经退行性疾病的发病或改善其症状。在此,我们展示了在生化分析和细胞培养中对刺激蛋白酶体非常有效的拟肽。它们在人血浆中稳定,并且能够穿透细胞膜。这些激活剂显示出增强α-突触核蛋白和tau的h20S降解的能力,其聚集体分别参与帕金森病和阿尔茨海默病的发展。这些拟肽对HEK293T细胞和原代海马细胞没有显示出细胞毒性。此外,在小鼠胚胎细胞模型中,这些化合物在减少海马神经元中磷酸化α-突触核蛋白聚集体的数量方面非常有效。