摘要
基因工程技术作为现代生物医学领域的重要工具，为药物研发带来了革命性变革。随着后基因组时代的到来，基因编辑、合成生物学和高通量测序技术的快速发展，药物研发在靶点发现、分子设计及疗效评估等方面取得了显著进展。本研究旨在探讨基因工程技术在药物研发中的最新应用及其潜在价值，通过系统分析CRISPR/Cas9、RNA干扰技术以及基因重组等方法在疾病模型构建、个性化治疗和新型疫苗开发中的作用，揭示其对药物研发效率和精准性的提升。研究表明，基因工程技术不仅能够加速药物靶点的验证过程，还为复杂疾病的机制解析提供了新视角。例如，基于CRISPR/Cas9的基因敲除技术已成功应用于肿瘤免疫治疗的靶点筛选，而基因重组技术则显著提高了抗体药物的生产效率和质量。此外，本研究首次提出将单细胞测序与基因编辑技术相结合的方法，用于优化药物筛选流程，从而大幅降低研发成本并缩短周期。综上所述，基因工程技术在药物研发中的应用正不断拓展，其创新性和实用性为未来生物医药产业的发展奠定了坚实基础，同时也为解决全球健康问题提供了新的可能方向。
关键词：基因工程技术；CRISPR/Cas9；药物研发；个性化治疗；单细胞测序
Abstract
Gene engineering technology, as a critical tool in modern biomedicine, has brought about revolutionary changes in drug development. With the advent of the post-genomic era and the rapid advancement of gene editing, synthetic biology, and high-throughput sequencing technologies, significant progress has been made in drug development in terms of target discovery, molecular design, and efficacy evaluation. This study aims to explore the latest applications and potential value of gene engineering technology in drug development by systematically analyzing the roles of CRISPR/Cas9, RNA interference, and gene recombination methods in disease model construction, personalized treatment, and novel vaccine development, thereby revealing their contributions to enhancing the efficiency and precision of drug development. The findings indicate that gene engineering technology not only accelerates the validation process of drug targets but also provides new perspectives for deciphering the mechanisms of complex diseases. For instance, CRISPR/Cas9-based gene knockout technology has been successfully applied to target screening in tumor immunotherapy, while gene recombination technology has significantly improved the production efficiency and quality of antibody drugs. Furthermore, this study proposes for the first time a method combining single-cell sequencing with gene editing technology to optimize drug screening procedures, substantially reducing development costs and shortening timelines. In conclusion, the application of gene engineering technology in drug development continues to expand, and its innovation and practicality lay a solid foundation for the future development of the biopharmaceutical industry while offering new possible directions for addressing global health challenges.
Keywords：Gene Engineering Technology; Crispr/Cas9; Drug Development; Personalized Treatment; Single-Cell Sequencing
目 录
摘要 I
Abstract II
一、绪论 1
(一) 基因工程技术与药物研发的背景意义 1
(二) 国内外研究现状分析 1
(三) 本文研究方法与技术路线 2
二、基因编辑技术在药物靶点发现中的应用 2
(一) CRISPR/Cas9技术原理及优势 2
(二) 基因编辑在疾病模型构建中的作用 3
(三) 靶点验证与药物筛选的新策略 3
三、基因重组技术在生物制药中的进展 4
(一) 重组蛋白药物的研发历程 4
(二) 单克隆抗体的基因工程优化 4
(三) 基因重组技术在疫苗开发中的应用 5
四、基因治疗技术在药物创新中的突破 5
(一) 基因载体技术的发展与挑战 5
(二) 基因疗法在遗传病治疗中的应用 6
(三) 基因治疗与传统药物的结合前景 7
结 论 8
参考文献 9
