Khatri Nirav I, Rathi Mohan N, Kolte Atul A, Kore Girish G, Lalan Manisha S, Trehan Sonia, Misra Ambikanandan R
Pharmacy Department, Faculty of Technology and Engineering, The Maharaja Sayajirao University of Baroda, Kalabhavan, Vadodara - 390 001, Gujarat state, India.
Recent Pat Drug Deliv Formul. 2012 Apr 1;6(1):45-65. doi: 10.2174/187221112799219116.
siRNA inhibits protein expression by degrading complementary mRNA sequence and hence, it is widely applicable for the treatment of various diseases where single or multiple gene knock down is necessary. Due to the severity and lethality of pulmonary diseases, siRNA has been focused for improved health in these diseases. Pulmonary accumulation of siRNA can be achieved by different means like intranasal or inhalation administration or intratracheal route which is mainly utilized for in vivo animal studies. However, various pulmonary obstacles and intracellular barriers for siRNA transport challenge this novel therapeutic moiety. Researchers have utilized different viral and non-viral delivery vectors for intracellular delivery of siRNA to knock down target mRNA. The promise of RNA interference, mediated by siRNAs, has revolutionized the prospects for modulating gene expression as a way to achieve therapeutic aims in disease treatment. This review focuses on patents describing the siRNA delivery either in naked form or along with a single/multiple delivery vectors. Many inventors have shown promising results for pulmonary utilization of siRNA and more concentration on delivery system may make this genomic approach available to the clinics soon.
小干扰RNA（siRNA）通过降解互补mRNA序列来抑制蛋白质表达，因此，它广泛适用于各种需要单基因或多基因敲除的疾病治疗。由于肺部疾病的严重性和致死性，siRNA已成为改善这些疾病健康状况的研究重点。siRNA在肺部的蓄积可通过不同方式实现，如鼻内给药、吸入给药或气管内给药，气管内给药主要用于体内动物研究。然而，siRNA转运面临的各种肺部障碍和细胞内屏障对这种新型治疗药物构成了挑战。研究人员已利用不同的病毒和非病毒递送载体将siRNA递送至细胞内，以敲低靶mRNA。由siRNA介导的RNA干扰有望彻底改变通过调节基因表达来实现疾病治疗目标的前景。本综述重点关注描述以裸形式或与单一/多种递送载体一起递送siRNA的专利。许多发明者已展示出siRNA肺部应用的有前景的结果，更多地关注递送系统可能会使这种基因组方法很快应用于临床。
