摘 要
基因治疗作为精准医学的重要组成部分，为多种遗传性和获得性疾病提供了新的治疗思路。然而，传统基因递送系统存在靶向性差、免疫原性强和转染效率低等缺陷，限制了其临床应用。纳米载体凭借独特的物理化学性质，在提高基因递送效率、增强组织靶向性及降低免疫反应等方面展现出显著优势。本研究综述了近年来纳米载体在基因治疗领域的研究进展，重点探讨了无机纳米材料（如金纳米颗粒、介孔二氧化硅）、有机纳米材料（如脂质体、聚合物）以及复合纳米材料的设计合成及其生物学特性。通过对比不同纳米载体的理化性质、细胞摄取机制、体内分布规律和生物安全性评价，揭示了纳米载体在基因治疗中的作用机制。研究发现，功能化修饰后的纳米载体能够有效提高基因递送效率，延长药物半衰期，并实现对特定细胞或组织的选择性靶向。同时，针对当前面临的挑战，如载体稳定性、免疫逃逸能力、大规模制备工艺等问题进行了深入分析。研究表明，开发智能响应型纳米载体是未来发展方向之一，这类载体可根据微环境变化实现可控释放，从而提高治疗效果并减少副作用。此外，多学科交叉融合为解决纳米载体在基因治疗中遇到的问题提供了新思路，有望推动该领域取得更大突破。
关键词：基因治疗；纳米载体；靶向性
Abstract
Gene therapy, as a crucial component of precision medicine, offers novel therapeutic strategies for various genetic and acquired diseases. However, traditional gene delivery systems suffer from poor targeting, strong immunogenicity, and low transfection efficiency, which limit their clinical applications. Nanocarriers, leveraging their unique physicochemical properties, have demonstrated significant advantages in enhancing gene delivery efficiency, improving tissue targeting, and reducing immune responses. This review summarizes recent advances in the application of nanocarriers in gene therapy, focusing on the design synthesis and biological characteristics of inorganic nanomaterials (such as gold nanoparticles and mesoporous silica), organic nanomaterials (such as liposomes and polymers), and composite nanomaterials. By comparing the physicochemical properties, cellular uptake mechanisms, in vivo distribution patterns, and biosafety evaluations of different nanocarriers, this study elucidates the mechanisms of nanocarriers in gene therapy. It was found that functionalized nanocarriers can effectively enhance gene delivery efficiency, prolong drug half-life, and achieve selective targeting of specific cells or tissues. Additionally, this paper provides an in-depth analysis of current challenges such as carrier stability, immune evasion capability, and large-scale manufacturing processes. Research indicates that developing intelligent responsive nanocarriers is one of the future directions, where these carriers can achieve controlled release in response to microenvironmental changes, thereby improving therapeutic efficacy and reducing side effects. Moreover, interdisciplinary integration offers new approaches to address issues encountered by nanocarriers in gene therapy, potentially driving greater breakthroughs in this field.
Keywords：Gene Therapy；NanoCarrier；Targeting；Gene Delivery Efficiencys
目 录
摘 要 I
Abstract II
引 言 1
第一章 纳米载体的基因递送机制 2
1.1 基因治疗的基本原理 2
1.2 纳米载体的结构与特性 2
1.3 递送机制及其优化策略 3
第二章 纳米载体在体内的行为 4
2.1 血液循环中的稳定性 4
2.2 细胞摄取与内吞途径 4
2.3 免疫反应及生物分布 5
第三章 纳米载体的应用进展 7
3.1 遗传疾病治疗应用 7
3.2 肿瘤靶向基因治疗 7
3.3 慢性病基因干预研究 8
第四章 纳米载体面临的挑战 10
4.1 安全性评估与监管 10
4.2 技术瓶颈与解决方案 10
4.3 临床转化的现实障碍 11
结 论 13
参考文献 14
致 谢 15
