Department of Molecular Biology and Biotechnology & Department of Biophysics, Biosciences Institute-IB, (UFRGS), Porto Alegre CEP 91501-970, RS, Brazil.
Center for Biotechnology, Universidade Federal do Rio Grande do Sul (UFRGS), Porto Alegre CEP 91501-970, RS, Brazil.
Int J Mol Sci. 2019 Mar 15;20(6):1322. doi: 10.3390/ijms20061322.
Intrinsically disordered proteins (IDPs) do not have rigid 3D structures, showing changes in their folding depending on the environment or ligands. Intrinsically disordered proteins are widely spread in eukaryotic genomes, and these proteins participate in many cell regulatory metabolism processes. Some IDPs, when aberrantly folded, can be the cause of some diseases such as Alzheimer's, Parkinson's, and prionic, among others. In these diseases, there are modifications in parts of the protein or in its entirety. A common conformational variation of these IDPs is misfolding and aggregation, forming, for instance, neurotoxic amyloid plaques. In this review, we discuss some IDPs that are involved in neurodegenerative diseases (such as beta amyloid, alpha synuclein, tau, and the "IDP-like" PrP), cancer (p53, c-Myc), and diabetes (amylin), focusing on the structural changes of these IDPs that are linked to such pathologies. We also present the IDP modulation mechanisms that can be explored in new strategies for drug design. Lastly, we show some candidate drugs that can be used in the future for the treatment of diseases caused by misfolded IDPs, considering that cancer therapy has more advanced research in comparison to other diseases, while also discussing recent and future developments in this area of research. Therefore, we aim to provide support to the study of IDPs and their modulation mechanisms as promising approaches to combat such severe diseases.
无规卷曲蛋白质(IDPs)没有刚性的 3D 结构,其折叠会根据环境或配体的变化而改变。无规卷曲蛋白质在真核基因组中广泛存在,这些蛋白质参与许多细胞调节代谢过程。一些 IDPs 在异常折叠时可能成为某些疾病的原因,如阿尔茨海默病、帕金森病和朊病毒病等。在这些疾病中,蛋白质的部分或全部发生了修饰。这些 IDPs 的常见构象变化是错误折叠和聚集,形成例如神经毒性淀粉样斑块。在这篇综述中,我们讨论了一些参与神经退行性疾病(如β淀粉样蛋白、α 突触核蛋白、tau 和“IDP 样”PrP)、癌症(p53、c-Myc)和糖尿病(淀粉样蛋白)的 IDPs,重点介绍了这些 IDPs 的结构变化与这些病理相关联的情况。我们还介绍了 IDP 调节机制,这些机制可以在药物设计的新策略中进行探索。最后,我们展示了一些候选药物,这些药物可以在未来用于治疗由错误折叠的 IDPs 引起的疾病,考虑到与其他疾病相比,癌症治疗的研究更为先进,同时还讨论了该研究领域的最新和未来发展。因此,我们旨在为 IDPs 及其调节机制的研究提供支持,这些机制是对抗这些严重疾病的有前途的方法。