Wang Chenchen, Zhou Qinghui
Allergan, Inc. Irvine, CA 92612, USA.
Recent Pat Drug Deliv Formul. 2012 Apr 1;6(1):19-30. doi: 10.2174/187221112799219080.
Small interfering RNA (siRNA) is a small, double-stranded RNA molecule that can suppress expression of specific genes. It will be a promising therapeutic agent for the treatment of viral and genetic diseases, such as HIV infection and cancer. However, it is extremely challenging to systemically deliver siRNA to the intended sites of action due to a variety of extracellular and intracellular barriers. Over the past decade, many novel patents have been developed to improve delivery efficiency and therapeutic efficacy of siRNA. The features of the carriers and technologies for systemic delivery of siRNA in these patents are summarized, and their clinical applications are discussed in this review.
小干扰RNA（siRNA）是一种小的双链RNA分子，能够抑制特定基因的表达。它将成为治疗病毒和遗传疾病（如HIV感染和癌症）的一种有前景的治疗药物。然而，由于多种细胞外和细胞内屏障，将siRNA全身递送至预期作用位点极具挑战性。在过去十年中，已经开发了许多新型专利来提高siRNA的递送效率和治疗效果。本文综述总结了这些专利中用于siRNA全身递送的载体和技术的特点，并讨论了它们的临床应用。
