Exploring the microRNAs and aptamers for their therapeutic role as biological drugs has expanded the horizon of its applicability against various human diseases, explicitly targeting the genetic materials. RNA-based therapeutics are widely being explored for the treatment and diagnosis of multiple diseases, including neurodegenerative disorders (NDD). Latter includes microRNA, aptamers, ribozymes, and small interfering RNAs (siRNAs), which control the gene expression mainly at the transcriptional strata. One RNA transcript translates into different protein types; hence, therapies targeted at the transcriptional sphere may have prominent and more extensive effects than alternative therapeutics. Unlike conventional gene therapy, RNAs, upon delivery, can either altogether abolish or alter the synthesis of the protein of interest, therefore, regulating their activities in a controlled and diverse manner. NDDs like Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, multiple sclerosis, Prion disease, and others are characterized by deposition of misfolded protein such as amyloid-ß, tau, α-synuclein, huntingtin and prion proteins. Neuroinflammation, one of the perquisites for neurodegeneration, is induced during neurodegenerative pathogenesis. In this review, we discuss microRNAs and aptamers' role as two different RNA-based approaches for their unique ability to regulate protein production at the transcription level, hence offering many advantages over other biologicals. The microRNA acts either by alleviating the malfunctioning RNA expression or by working as a replacement to lost microRNA. On the contrary, aptamer act as a chemical antibody and forms an aptamer-target complex.

探索 microRNA 和适体作为生物药物的治疗作用，扩大了其针对各种人类疾病的适用范围，明确针对遗传物质。基于 RNA 的疗法正在广泛探索用于治疗和诊断多种疾病，包括神经退行性疾病 (NDD)。后者包括 microRNA、适体、核酶和小干扰 RNA (siRNA)，它们主要在转录层控制基因表达。一种RNA转录物翻译成不同的蛋白质类型；因此，针对转录领域的疗法可能比替代疗法具有更显着和更广泛的效果。与传统的基因治疗不同，RNA 在递送后可以完全废除或改变目标蛋白质的合成，因此以受控和多样化的方式调节其活性。阿尔茨海默病、帕金森病、亨廷顿病、多发性硬化症、朊病毒病等 NDD 的特点是错误折叠蛋白的沉积，如淀粉样蛋白-β、tau、α-突触核蛋白、亨廷顿蛋白和朊病毒蛋白。神经炎症是神经退行性病变的先决条件之一，是在神经退行性发病过程中诱发的。在这篇综述中，我们讨论了 microRNA 和适体作为两种不同的基于 RNA 的方法的作用，因为它们在转录水平上调节蛋白质生产的独特能力，因此比其他生物制品具有许多优势。 microRNA 的作用是减轻 RNA 表达故障或替代丢失的 microRNA。相反，适体充当化学抗体并形成适体-靶标复合物。