Abstract：
RNA therapeutics have emerged as potential treatments for genetic disorders, infectious diseases, and cancer. RNA delivery to target cells for efficient therapeutic applications remains challenging due to instability and poor uptake. Polymeric nanoparticulate delivery systems offer stability, protection, and controlled release. These systems shield RNA from degradation, enabling efficient uptake and extended circulation. Various polymeric nanoparticle platforms have been explored, including lipid-based nanoparticles, polymeric micelles, dendrimers, and polymer-drug conjugates. This review outlines recent breakthroughs of recent advances, design principles, characterization techniques, and performance evaluation of these delivery systems. It highlights their potential in translating preclinical studies into clinical applications. Additionally, the review discusses the application of polymeric nanoparticles in ophthalmic drug delivery, particularly for medications that dissolve poorly in water, and the progress made in siRNA-based therapies for viral infections, autoimmune diseases, and cancers. SiRNA holds great promise for precision medicine and therapeutic intervention, with the ability to target specific genes and modulate disease-associated pathways. The versatility and potency of siRNA-based drugs offer a broader scope for therapeutic intervention compared to traditional biological drugs. As research in RNA therapeutics continues to advance, these technologies hold tremendous potential to revolutionize the treatment of various diseases and improve patient outcomes.
摘要：
RNA疗法已经成为遗传疾病的潜在治疗方法，传染病,和癌症。由于不稳定性和摄取差,用于有效治疗应用的RNA递送至靶细胞仍然具有挑战性。聚合物纳米颗粒递送系统提供稳定性，保护,和控制释放。这些系统保护RNA免于降解，使有效的摄取和延长的循环。已经探索了各种聚合物纳米颗粒平台，包括基于脂质的纳米颗粒，聚合物胶束，树枝状聚合物,和聚合物-药物缀合物。这篇综述概述了最新进展的最新突破，设计原则,表征技术，和这些交付系统的性能评估。它突出了他们在将临床前研究转化为临床应用方面的潜力。此外,该综述讨论了聚合物纳米颗粒在眼科药物递送中的应用，特别是对于水溶性差的药物，以及基于siRNA的病毒感染疗法的进展，自身免疫性疾病，和癌症。SiRNA在精准医学和治疗干预方面具有广阔的前景。具有靶向特定基因和调节疾病相关途径的能力。与传统生物药物相比，基于siRNA的药物的多功能性和效力为治疗干预提供了更广泛的范围。随着RNA疗法研究的不断深入,这些技术在彻底改变各种疾病的治疗和改善患者预后方面具有巨大潜力。
