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基因治疗中的免疫调节策略:克服免疫障碍并提高疗效。

Immune Modulation Strategies in Gene Therapy: Overcoming Immune Barriers and Enhancing Efficacy.

作者信息

Amsaveni Sivaprakasam, Radha Mahendran, Chandrasekaran Vidhya, Chanchal Dilip Kumar, Mathew Sojomon, Sharma Mukesh Chandra, Shiekmydeen Jailani, Ali Syed Salman

机构信息

Department of Bioinformatics, School of Life Sciences, Vels Institute of Science, Technology and Advanced Studies (VISTAS), Pallavaram 600117, Chennai, Tamilnadu, India.

Department of Primary Processing Storage and Handling, NIFTEM-Thanjavur, Thanjavur 613005, Tamil Nadu, India.

出版信息

Curr Gene Ther. 2025;25(4):374-393. doi: 10.2174/0115665232305409240918040639.

Abstract

The immune system presents significant obstacles to gene therapy, which has limited its use in treating many illnesses. New approaches are needed to overcome these problems and improve the effectiveness of gene therapy. This study explores several techniques to immune regulation within gene therapy, a cutting-edge discipline that aims to optimise results by fine-tuning the immune response. We cover new ways to control the immune system and deliver therapeutic genes just where they are needed, including influencing immunological checkpoints, causing immunotolerance, and making smart use of immunomodulatory drugs. In addition, the study provides insight into new developments in the design of less immunogenic gene delivery vectors, which allow for the extension of transgene expression with minimal adverse immune reactions. In order to maximise the efficacy of gene-based therapies, this review analyses these novel approaches and gives a thorough overview of the present state of the art by addressing obstacles and pointing the way toward future developments in immune regulation. Not only does their integration provide new opportunities for the creation of safer and more effective gene treatments, but it also contains the key to overcome current obstacles.

摘要

免疫系统给基因治疗带来了重大障碍,这限制了其在治疗多种疾病中的应用。需要新的方法来克服这些问题并提高基因治疗的有效性。本研究探索了基因治疗中免疫调节的几种技术,这是一个前沿学科,旨在通过微调免疫反应来优化治疗效果。我们涵盖了控制免疫系统以及将治疗性基因精准递送至所需部位的新方法,包括影响免疫检查点、诱导免疫耐受以及巧妙利用免疫调节药物。此外,该研究还深入探讨了低免疫原性基因递送载体设计方面的新进展,这些载体能够在产生最小不良免疫反应的情况下延长转基因表达。为了使基于基因的治疗效果最大化,本综述分析了这些新方法,并通过阐述障碍和指明免疫调节未来发展方向,全面概述了当前的技术现状。它们的整合不仅为创造更安全、更有效的基因治疗提供了新机会,还包含克服当前障碍的关键。

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