面向精准调控肿瘤细胞微环境的核酸递送系统的研究

基因治疗被认为是攻克威胁人类健康的一系列重大疾病（如癌症、心脑血管疾病等）最具应用前景的治疗手段之一。本申请希望将级联催化的概念引入到基因治疗递送系统的设计中，构建可调节癌细胞微环境的阳离子递送载体，设计表达后可与微环境中活性物质相互作用的功能核酸，利用递送的细胞微环境调控物质与核酸表达出的蛋白实现肿瘤细胞内级联催化，最终构建可用于精准调控肿瘤细胞微环境的功能核酸递送系统。探讨调节细胞微环境与核酸表达在级联调控肿瘤细胞微环境中的作用机制，达到一体递送，分步调控的治疗效果。本项目有望为构建多功能新型医用递送载体提供新思路。

Gene therapy is considered as a promising therapeutic method to overcome a series of major diseases that threaten human health, such as, cancer, cerebrovascular diseases. We seek to introduce the concept of cascade catalysis into the design of gene therapy delivery systems that can regulate the cellular microenvironment and express functional proteins which could be interacted with bio-activators in the microenvironment. This project attempts to explore the mechanism of regulating Cellular microenvironment and proteins expression in synergistic effect of cancer therapy. Finally, a series of genetically cascade therapy systems will be developed. This proposal is expected to provide a new method for the designation of new generation of multifunctional delivery carriers for biomedical applications.