Shobhaben Pratapbhai Patel School of Pharmacy & Technology Management, SVKM's NMIMS (Deemed to be University), Mumbai, 400056, India.
Department of Pharmaceutical Sciences, Mohanlal Sukhadia University, Udaipur, Rajasthan, 313001, India.
AAPS PharmSciTech. 2023 Aug 11;24(6):170. doi: 10.1208/s12249-023-02629-1.
Since the ground-breaking discovery of RNA interference (RNAi), scientists have made significant progress in the field of small interfering RNA (siRNA) treatments. Due to severe barriers to the therapeutic application of siRNA, nanoparticle technologies for siRNA delivery have been designed. For pathological circumstances such as viral infection, toxic RNA abnormalities, malignancies, and hereditary diseases, siRNAs are potential therapeutic agents. However, systemic administration of siRNAs in vivo remains a substantial issue due to a lack of "drug-likeness" (siRNA are relatively larger than drugs and have low hydrophobicity), physiological obstacles, and possible toxicities. This write-up covers important accomplishment in the field of clinical trials and patents specially based of siRNAs using targeting viruses. Furthermore, it offers deep insight of nanoparticle applied for siRNA delivery and strategies to improve the effectiveness of antivirals.
自 RNA 干扰 (RNAi) 的突破性发现以来，科学家在小干扰 RNA (siRNA) 治疗领域取得了重大进展。由于 siRNA 的治疗应用存在严重障碍，因此设计了用于 siRNA 递送的纳米颗粒技术。对于病毒感染、毒性 RNA 异常、恶性肿瘤和遗传性疾病等病理情况，siRNA 是潜在的治疗剂。然而，由于缺乏“药物样特性”（siRNA 相对较大且疏水性低）、生理障碍和可能的毒性，siRNA 在体内的全身给药仍然是一个重大问题。本文涵盖了临床试验和专利领域的重要成果，特别是基于靶向病毒的 siRNAs。此外，它还深入探讨了用于 siRNA 递送的纳米颗粒以及提高抗病毒药物效果的策略。
