Nanomedicine Research Centre, Department of Pharmaceutics, Indo-Soviet Friendship, College of Pharmacy, Moga, Punjab, India.
Curr Gene Ther. 2010 Apr;10(2):139-55. doi: 10.2174/156652310791111010.
Small interfering RNAs (siRNA) are one of the most recent additions used to silence gene expression. At present siRNA is the most extensively used gene-silencing technique over other nucleic-acid based approaches to treat disease including cancer, hepatitis, respiratory disease, cardiovascular diseases, neuronal disease and autoimmune disease. However, systemic delivery of siRNA in vivo, remains to be the biggest challenge to be overcome. Various strategies have been developed to deliver siRNA efficiently into target cell such as chemical modification of siRNA, physical strategies, viral and non viral-vectors mediated delivery. Among all the approaches non viral vectors including lipoplexes and polyplexes were found to be most successful which have been reviewed in this article. Further therapeutic applications of RNAi have also been briefly reviewed.
小干扰 RNA(siRNA)是最近用于沉默基因表达的方法之一。目前,与其他基于核酸的方法相比,siRNA 是治疗疾病(包括癌症、肝炎、呼吸道疾病、心血管疾病、神经疾病和自身免疫性疾病)最广泛使用的基因沉默技术。然而,siRNA 的体内系统给药仍然是需要克服的最大挑战。已经开发了各种策略来将 siRNA 有效地递送到靶细胞中,例如 siRNA 的化学修饰、物理策略、病毒和非病毒载体介导的递药。在所有方法中,非病毒载体(包括脂质体和聚合物)被发现是最成功的,本文对此进行了综述。本文还简要综述了 RNAi 的进一步治疗应用。