为了使基因治疗能够真正替代常规治疗方法，研发安全、靶向、高效的基因载体至关重要。本课题设计以N-琥珀酰壳聚糖为纳米基因载体材料，利用叶酸及小分子多肽Tat修饰N-琥珀酰壳聚糖，构建具有长循环特性、肿瘤靶向性以及跨膜转运功能的Tat-N-Suc-F-Chi/psiRNA自组装纳米基因传递系统，用于实现非小细胞肺癌的基因治疗。该纳米基因载体能特异性地被靶细胞识别并结合，可以将基因安全地载入靶细胞，快速定位到细胞核，增强基因摄取和表达，降低毒副作用，实现纳米基因传递系统的靶向性和高效转染性，从而实现RNAi对非小细胞肺癌的治疗作用。本课题构建Tat-N-Suc-F-Chi纳米基因传递系统，将纳米肿瘤靶向传递系统与RNAi相结合用于非小细胞肺癌的治疗是肿瘤基因治疗研究领域一次新的尝试，可望拓宽基因载体发展方向，为非小细胞肺癌的基因治疗研究奠定基础，提供新的技术平台。

In order to make gene therapy can be a substitute for conventional treatment methods, research and development of security, targeting, efficient gene delivery vectors is crucial. PsiRNA loaded folate and Tat mediated self-assembly nanoparticles with a long-circulating, tumor targeting and transmembrane　transport function as gene delivery system were constructed to achieve non small cell lung cancer gene therapy in this program. The nanoparticles gene carriers can be specifically recognized and binded by target cell, gene can be carried into target cells, rapidly positioned to the nucleus, enhanced uptake and expression of gene, reduced toxic and side effect, to achieve targeted and efficient transfection of nano gene delivery system and the therapy of RNAi on non small cell lung cancer . Nano tumor targeting delivery system combined with RNAi used for non small cell lung cancer therapy is a new attempt in tumor gene therapy research field, to build the foundation and provide a new technical platform for the research of non small cell lung cancer gene therapy, expected to widen the gene vector direction.