Alla Sai Santhosha Mrudula, Shah Dhruv Jayeshkumar, Meyur Shourya, Agarwal Pahel, Alla Deekshitha, Moraboina Sai Lokesh, Ghadvaje Gayatri Vijay, Bayeh Ruth Getaneh, Malireddi Aparna, Shajan Tess, Vineetha Bodipudi, Sai Prudhvi Thalvayapati, Biziyaremye Patrick
Department of General Medicine, Andhra Medical College, Visakhapatnam, India.
Department of General Medicine, Massachusetts College of Pharmacy and Health Sciences (MCPHS), Boston, MA, USA.
J Exp Pharmacol. 2025 May 28;17:249-267. doi: 10.2147/JEP.S521579. eCollection 2025.
RNA interference (RNAi) therapy represents an evolving advancement in the management of dyslipidemia. One prominent form of RNAi therapy is small interfering RNA (siRNA), which has emerged as a promising therapeutic strategy. This study aims to critically analyze the efficacy and safety of siRNA in the treatment of dyslipidemia.
PubMed, Scopus, and Web of Science servers were used to conduct a systematic search in compliance with the PRISMA guidelines.
A total of 20 studies with 6651 participants were included in the analysis. The drugs used in the studies were. Inclisiran led to a notable 44.09% reduction in LDL and 37.5% in apolipoprotein levels among individuals with hypercholesterolemia. In hyperlipoproteinemia(a), therapies like Lepodisiran and Olpasiran achieved a 75.69% drop in apolipoproteins and 16.25% in LDL. For hypertriglyceridemia, agents such as ARO-APOC3 and Plozasiran showed over 50% reductions in both VLDL and triglycerides. In mixed hyperlipidemia and chylomicronemia, Plozasiran significantly reduced triglycerides by up to 79% and apolipoproteins by 87.5%. The 5 most common adverse effects reported were nasopharyngitis, diabetes mellitus (including new-onset diabetes mellitus and worsening diabetes mellitus), injection site adverse effects, back pain, and hypertension.
In conclusion, the benefits of siRNA therapy in dyslipidemia management appear to outweigh its potential drawbacks, demonstrating promising efficacy and safety profiles. However, further research is necessary to fully understand its long-term effects and optimize its therapeutic potential.
RNA干扰（RNAi）疗法是血脂异常管理领域不断发展的一项进步。RNAi疗法的一种主要形式是小干扰RNA（siRNA），它已成为一种有前景的治疗策略。本研究旨在严格分析siRNA治疗血脂异常的疗效和安全性。
按照PRISMA指南，使用PubMed、Scopus和Web of Science数据库进行系统检索。
分析共纳入20项研究，涉及6651名参与者。研究中使用的药物有……。Inclisiran使高胆固醇血症患者的低密度脂蛋白（LDL）显著降低44.09%，载脂蛋白水平降低37.5%。在高脂蛋白血症（a）中，Lepodisiran和Olpasiran等疗法使载脂蛋白降低75.69%，LDL降低16.25%。对于高甘油三酯血症，ARO-APOC3和Plozasiran等药物使极低密度脂蛋白（VLDL）和甘油三酯均降低超过50%。在混合性高脂血症和乳糜微粒血症中，Plozasiran使甘油三酯显著降低高达79%，载脂蛋白降低87.5%。报告的5种最常见不良反应为鼻咽炎、糖尿病（包括新发糖尿病和糖尿病恶化）、注射部位不良反应、背痛和高血压。
总之，siRNA疗法在血脂异常管理中的益处似乎超过其潜在弊端，显示出有前景的疗效和安全性。然而，需要进一步研究以充分了解其长期影响并优化其治疗潜力。
