Singh Yashpreet, Singh Aniket, Geoffrey Priscar, Chaitanya Motamarri Venkata Naga Lalitha, Kaur Jaskiran
School of Pharmaceutical Sciences, Lovely Professional University, Jalandhar, Phagwara GT Road, Punjab, 144411, India.
Curr Pharm Des. 2025;31(22):1755-1766. doi: 10.2174/0113816128331823241121055205.
Rheumatoid Arthritis (RA) is a chronic autoimmune disease that prominently affects the joints of the body, including one or more types, such as wrists, knees, fingers, and toes. This condition is characterized by specific auto-immune responses, i.e., inflammation, swelling, stiffness, and pain in the body's joints. This results in partial or complete immobility. Several treatment options are available in the pharmaceutical market, commonly immunosuppressants, anti-inflammatory drugs, corticosteroids, anti-antibody drugs, etc. However, such drug treatments provide only symptomatic relief and lack long-term therapeutic efficacy. Thus, there is a need to develop an alternative treatment option that potentially can cure this disease. Recently, researchers have shifted their focus to targeting the root cause involved in the pathogenesis of RA by designing nucleic-acid- based molecules. With this objective, we have provided this review in which we have explored the nucleic acid-based techniques used to treat RA. Various molecular and genetic methods, including antisense oligonucleotides, small interfering RNA (siRNA), CRISPR-Cas9 gene editing, RNA-based vaccines, microRNA (miRNA) therapeutics, and epigenetic modifications are used to target the mechanisms underlying RA pathogenesis. Through the extensive evaluation of clinical and preclinical studies, we reported nucleic acid-based therapy's clinical efficacy, safety, and therapeutic benefits. In addition, this study states the associated pharmacokinetic challenges followed by approaches to overcome them. Nucleic acid-based therapies are susceptible to degradation by nucleases in the body and are not efficiently absorbed due to their considerable molecular weight. Specialized carriers like nanoparticles are employed for their effective delivery. Nucleic acid-based therapies hold immense potential for achieving more effective, less toxic, and patient-centric management of RA, offering hope for improved outcomes and an enhanced quality of life for individuals living with this challenging autoimmune condition.
类风湿性关节炎(RA)是一种慢性自身免疫性疾病,主要影响身体关节,包括手腕、膝盖、手指和脚趾等一种或多种类型。这种疾病的特征是特定的自身免疫反应,即身体关节的炎症、肿胀、僵硬和疼痛。这会导致部分或完全无法活动。制药市场上有几种治疗选择,常见的有免疫抑制剂、抗炎药、皮质类固醇、抗抗体药物等。然而,这类药物治疗仅能缓解症状,缺乏长期治疗效果。因此,需要开发一种可能治愈这种疾病的替代治疗方案。最近,研究人员已将重点转向通过设计基于核酸的分子来针对RA发病机制中的根本原因。出于这个目的,我们提供了这篇综述,其中探讨了用于治疗RA的基于核酸的技术。各种分子和基因方法,包括反义寡核苷酸、小干扰RNA(siRNA)、CRISPR-Cas9基因编辑、基于RNA的疫苗、微小RNA(miRNA)疗法和表观遗传修饰,都被用于针对RA发病机制的潜在机制。通过对临床和临床前研究的广泛评估,我们报告了基于核酸疗法的临床疗效、安全性和治疗益处。此外,本研究阐述了相关的药代动力学挑战以及克服这些挑战的方法。基于核酸的疗法易受体内核酸酶的降解,并且由于其分子量较大而不能有效吸收。像纳米颗粒这样的专业载体被用于其有效递送。基于核酸的疗法在实现更有效、毒性更小且以患者为中心的RA管理方面具有巨大潜力,为改善患有这种具有挑战性的自身免疫性疾病的个体的治疗结果和提高生活质量带来了希望。