Rai Sachchida Nand, Tiwari Neeraj, Singh Payal, Mishra Divya, Singh Anurag Kumar, Hooshmandi Etrat, Vamanu Emanuel, Singh Mohan P
Centre of Biotechnology, University of Allahabad, Prayagraj, India.
Faculty of Biosciences, Institute of Biosciences and Technology, Shri Ramswaroop Memorial University, Barabanki, India.
Front Neurosci. 2021 Dec 14;15:777347. doi: 10.3389/fnins.2021.777347. eCollection 2021.
Autophagy is an important cellular self-digestion and recycling pathway that helps in maintaining cellular homeostasis. Dysregulation at various steps of the autophagic and endolysosomal pathway has been reported in several neurodegenerative disorders such as Alzheimer's disease (AD), Parkinson's disease (PD), and Huntington disease (HD) and is cited as a critically important feature for central nervous system (CNS) proteostasis. Recently, another molecular target, namely transcription factor EB (TFEB) has been explored globally to treat neurodegenerative disorders. This TFEB, is a key regulator of autophagy and lysosomal biogenesis pathway. Multiple research studies suggested therapeutic potential by targeting TFEB to treat human diseases involving autophagy-lysosomal dysfunction, especially neurodegenerative disorders. A common observation involving all neurodegenerative disorders is their poor efficacy in clearing and recycle toxic aggregated proteins and damaged cellular organelles due to impairment in the autophagy pathway. This dysfunction in autophagy characterized by the accumulation of toxic protein aggregates leads to a progressive loss in structural integrity/functionality of neurons and may even result in neuronal death. In recent years TFEB, a key regulator of autophagy and lysosomal biogenesis, has received considerable attention. It has emerged as a potential therapeutic target in numerous neurodegenerative disorders like AD and PD. In various neurobiology studies involving animal models, TFEB has been found to ameliorate neurotoxicity and rescue neurodegeneration. Since TFEB is a master transcriptional regulator of autophagy and lysosomal biogenesis pathway and plays a crucial role in defining autophagy activation. Studies have been done to understand the mechanisms for TFEB dysfunction, which may yield insights into how TFEB might be targeted and used for the therapeutic strategy to develop a treatment process with extensive application to neurodegenerative disorders. In this review, we explore the role of different transcription factor-based targeted therapy by some natural compounds for AD and PD with special emphasis on TFEB.
自噬是一种重要的细胞自我消化和循环利用途径,有助于维持细胞内稳态。在阿尔茨海默病(AD)、帕金森病(PD)和亨廷顿病(HD)等多种神经退行性疾病中,自噬和内溶酶体途径的各个步骤均出现失调,这被认为是中枢神经系统(CNS)蛋白质稳态的一个至关重要的特征。最近,另一个分子靶点,即转录因子EB(TFEB)已在全球范围内被探索用于治疗神经退行性疾病。这个TFEB是自噬和溶酶体生物发生途径的关键调节因子。多项研究表明,靶向TFEB治疗涉及自噬 - 溶酶体功能障碍的人类疾病,尤其是神经退行性疾病具有治疗潜力。所有神经退行性疾病的一个共同现象是,由于自噬途径受损,它们在清除和循环有毒聚集蛋白及受损细胞器方面效果不佳。这种以有毒蛋白质聚集体积累为特征的自噬功能障碍会导致神经元结构完整性/功能逐渐丧失,甚至可能导致神经元死亡。近年来,TFEB作为自噬和溶酶体生物发生的关键调节因子受到了广泛关注。它已成为AD和PD等多种神经退行性疾病的潜在治疗靶点。在各种涉及动物模型的神经生物学研究中,已发现TFEB可改善神经毒性并挽救神经退行性变。由于TFEB是自噬和溶酶体生物发生途径的主要转录调节因子,在定义自噬激活中起关键作用。人们已经开展研究以了解TFEB功能障碍的机制,这可能有助于深入了解如何靶向TFEB并将其用于治疗策略,从而开发出广泛应用于神经退行性疾病的治疗方法。在本综述中,我们探讨了一些天然化合物基于不同转录因子的靶向治疗对AD和PD的作用,特别强调了TFEB。