AAPS PharmSciTech ( IF 3.4 ) Pub Date : 2023-08-11 , DOI: 10.1208/s12249-023-02629-1
Krittika Chatterjee 1 , Sagheerah Lakdawala 1 , Sheikh Shahnawaz Quadir 2 , Dinesh Puri 3 , Dinesh Kumar Mishra 4 , Garima Joshi 2 , Sanjay Sharma 1 , Deepak Choudhary 2
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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.
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基于 siRNA 的提高抗病毒药物有效性的新型治疗策略:见解
自从RNA干扰(RNAi)的突破性发现以来,科学家们在小干扰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、有毒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“药物相似性”(siRNA比药物相对较大且疏水性较低)、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。这篇文章涵盖了特别基于使用靶向病毒的 siRNA 的临床试验和专利领域的重要成就。此外,它还提供了应用于 siRNA 递送的纳米颗粒的深入见解以及提高抗病毒药物有效性的策略。