基于PAsp(DET)的新型基因递送体系的制备及其应用于肿瘤基因治疗的基础研究
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随着基因技术的发展，基因治疗开始应用于多种疾病的治疗，如遗传性疾病、免疫缺陷性疾病、恶性肿瘤等。在肿瘤基因治疗中，将基因药物精准、高效导入靶细胞至关重要。因此需要选择合适的运输工具，即载体。目前常用的基因载体包括病毒载体及非病毒载体。非病毒基因载体相对于病毒载体具有低毒、低免疫原性及易于制备等优点，但转染效率相对较低，因此开发高效低毒的非病毒基因载体成为了研究的难点及热点。本研究论文旨在通过靶向配体修饰和利用肿瘤微环境响应材料等策略构建高效、低毒的基因递送系统，以期开发出具有可观运用前景及现实意义的、适用于肿瘤基因治疗的新型基因递送系统。
With the advancement of genetic technology, gene therapy has been applied to the treatment of various diseases, including genetic disorders, immunodeficiency diseases, malignant tumors, and others. In tumor gene therapy, it is critical to deliver gene drugs accurately and efficiently into target cells. Therefore, it is necessary to select appropriate delivery vehicles, namely vectors. Currently, commonly used gene vectors include viral vectors and non-viral vectors. Compared with viral vectors, non-viral gene vectors boast advantages such as low toxicity, low immunogenicity, and ease of preparation, but their transfection efficiency is relatively low. As a result, developing efficient and low-toxicity non-viral gene vectors has emerged as a key research focus and challenge. This study aims to construct efficient and low-toxicity gene delivery systems via strategies including targeting ligand modification and utilization of tumor microenvironment-responsive materials, with the objective of developing novel gene delivery systems with promising application prospects and practical significance that are suitable for tumor gene therapy.
