The identification of RNA interference (RNAi) has caused a growing interest in harnessing its potential in the treatment of different diseases. Modulation of dysregulated genes through targeting by RNAi represents a potential approach with which to alter the biological pathways at a post-transcriptional level, especially as it pertains to autoimmunity and malignancy. Short hairpin RNAs (shRNA), short interfering RNAs (siRNA), and microRNAs (miRNA) are mainly involved as effector mechanisms in the targeting of RNAi biological pathways. The manipulation and delivery of these molecules in an efficient way promotes the specificity and stability of RNAi-based systems, while minimizing the unwanted adverse reactions by the immune system and reducing cytotoxicity and off-target effects. Advances made to date in identifying the etiopathogenesis of autoimmune diseases has prompted the utilization of RNAi-based systems in vitro and in vivo. Future investigations aimed at deciphering the molecular basis of RNAi and optimizing the delivery of RNAi-based targeting systems will hopefully promote the applicability of such regulatory mechanisms and, ultimately, transfer the acquired knowledge from bench-to-bedside to ameliorate human diseases. In this review, we seek to clarify the potential of RNAi, with a focus on siRNAs, in designing therapeutics for potential treatment of human autoimmune disorders.

RNA 干扰 (RNAi) 的鉴定引起了人们对利用其治疗不同疾病的潜力的兴趣。通过 RNAi 靶向调节失调的基因代表了一种潜在的方法，可以在转录后水平上改变生物学途径，尤其是在与自身免疫和恶性肿瘤有关的情况下。短发夹 RNA (shRNA)、短干扰 RNA (siRNA) 和微 RNA (miRNA) 主要作为效应机制参与 RNAi 生物途径的靶向。以有效的方式操纵和传递这些分子可提高基于 RNAi 的系统的特异性和稳定性，同时最大限度地减少免疫系统不必要的不​​良反应并降低细胞毒性和脱靶效应。体外和体内。未来旨在破译 RNAi 的分子基础和优化基于 RNAi 的靶向系统的传递的研究将有望促进此类调节机制的适用性，并最终将获得的知识从工作台转移到床边以改善人类疾病。在这篇综述中，我们试图阐明 RNAi 的潜力，重点是 siRNA，在设计治疗人类自身免疫性疾病的潜在治疗方法方面。