Mete Rumela, Das Sourav, Saha Arindam, Roy Sukanta, Mondal Smritilekha, Bose Anirbandeep, Basu Biswajit, Elossaily Gehan M, Prajapati Bhupendra
TAAB Biostudy Services, Jadavpur, Kolkata, 700032, India.
Department of Pharmaceutics, School of Pharmacy, The Neotia University, Sarisa, West Bengal, 743368, India.
Mol Biotechnol. 2025 Mar 27. doi: 10.1007/s12033-025-01426-4.
Transgenesis, the introduction of foreign genetic material into the genome of an organism, has become a crucial and transformative technique in the realm of drug discovery. This review article provides a comprehensive overview of the integral role that transgenesis plays in the drug discovery process, with a specific focus on target identification and target validation. By examining the recent advancements and innovative approaches, this article aims to shed light on the importance of transgenesis in accelerating drug development. In the context of target identification, transgenesis has allowed for the creation of relevant disease models, enabling researchers to study the genetic and molecular basis of various disorders. The use of transgenic animals, such as mice and zebrafish, has facilitated the identification of potential drug targets by mimicking specific human disease conditions. This review also discusses emerging technologies, such as CRISPR-Cas9 and other genome editing tools, which have revolutionized the field of transgenesis. These technologies have enhanced the precision and efficiency of genetic manipulations in transgenic animals, making the creation of disease-relevant models more accessible and cost-effective. Moreover, integration of omics technologies, such as genomics, transcriptomics, proteomics, and metabolomics, has provided a holistic view of the molecular changes in transgenic models, further aiding in target identification and validation. This review emphasizes the importance of transgenesis in target identification and validation and underscores its vital role in shaping the future of drug discovery.
转基因技术,即将外源遗传物质引入生物体基因组的技术,已成为药物研发领域一项关键且具有变革性的技术。这篇综述文章全面概述了转基因技术在药物研发过程中所起的不可或缺的作用,特别聚焦于靶点识别和靶点验证。通过审视近期的进展和创新方法,本文旨在阐明转基因技术在加速药物开发方面的重要性。在靶点识别方面,转基因技术使得创建相关疾病模型成为可能,使研究人员能够研究各种疾病的遗传和分子基础。使用转基因动物,如小鼠和斑马鱼,通过模拟特定的人类疾病状况,有助于识别潜在的药物靶点。本综述还讨论了新兴技术,如CRISPR - Cas9和其他基因组编辑工具,这些技术彻底改变了转基因领域。这些技术提高了转基因动物基因操作的精度和效率,使创建与疾病相关的模型变得更容易且更具成本效益。此外,组学技术(如基因组学、转录组学、蛋白质组学和代谢组学)的整合提供了转基因模型中分子变化的整体视图,进一步辅助靶点识别和验证。本综述强调了转基因技术在靶点识别和验证中的重要性,并突显了其在塑造药物研发未来方面的关键作用。