Centre for the Environment, IIT Guwahati, Assam 781039, India; Symbol Discovery Ltd, Hyderabad 500046, India.
Centre for the Environment, IIT Guwahati, Assam 781039, India; Department of Chemistry, Indian Institute of Technology Guwahati, 781039, India.
Bioorg Med Chem Lett. 2023 Jul 15;91:129379. doi: 10.1016/j.bmcl.2023.129379. Epub 2023 Jun 16.
An increasing number of neurodegenerative diseases seem to be associated with protein misfolding that often leads to misfolded protein aggregates with a β-sheet conformation and accumulation in the brain which directly contributes to or modulates the associated pathology. Protein aggregation diseases like Huntington's disease results from the deposition of aggregated huntingtin proteins within the nucleus, transmissible prion encephalopathies occur due to extracellular deposition of pathogenic prion proteins whereas Alzheimer's disease from the accumulation of both extracellular β-amyloid and intracellular hyperphosphorylated tau protein aggregates. In the generalized purpose, we have taken the core sequence of amyloid-β (responsible for its aggregation) as the aggregating peptide (AP). Among the various emerging therapeutic approaches against aggregation-related degenerative diseases such as diminishing the monomeric precursor protein, inhibiting aggregation, or blocking aggregation-induced cellular toxicity pathways, we focussed on the strategy based on the inhibition of protein aggregation using rationally designed peptide inhibitors comprising both the recognition and β-breaking component in the sequence. The "O → N acyl migration" concept was used to form cyclic peptide in situ for the generation of a bent unit which may act as disruption moiety for the inhibition process. The kinetics of aggregation was characterized by various biophysical tools (ThT-assay, TEM, CD, and FTIR). Results implied that the designed inhibitor peptides (IP) might be valuable to inhibit all the related aggregated peptides.
越来越多的神经退行性疾病似乎与蛋白质错误折叠有关,这种错误折叠常常导致具有β-折叠构象的错误折叠蛋白聚集,并在大脑中积累,这直接导致或调节相关的病理学。像亨廷顿病这样的蛋白聚集疾病是由于核内聚集的亨廷顿蛋白沉积引起的,可传播的朊病毒脑病是由于细胞外致病性朊病毒蛋白的沉积引起的,而阿尔茨海默病则是由于细胞外β-淀粉样蛋白和细胞内过度磷酸化的 tau 蛋白聚集引起的。在广义上,我们将淀粉样-β(负责其聚集)的核心序列作为聚集肽(AP)。在针对与聚集相关的退行性疾病的各种新兴治疗方法中,如减少单体前体蛋白、抑制聚集或阻断聚集诱导的细胞毒性途径,我们专注于基于使用合理设计的肽抑制剂抑制蛋白质聚集的策略,这些抑制剂包含序列中的识别和β-断裂成分。“O→N 酰基迁移”概念用于在原位形成环肽,以生成弯曲单元,该单元可作为抑制过程的破坏部分。聚集的动力学通过各种生物物理工具(ThT 测定法、TEM、CD 和 FTIR)进行了表征。结果表明,设计的抑制剂肽(IP)可能对抑制所有相关的聚集肽都有价值。