基因疗法治疗胃肠道疾病的潜力:进展与未来展望。

IF 5.3 2区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL
Molecular Therapy Oncolytics Pub Date : 2023-08-18 eCollection Date: 2023-09-21 DOI:10.1016/j.omto.2023.08.007
Ning-Ning Yue, Hao-Ming Xu, Jing Xu, Min-Zheng Zhu, Yuan Zhang, Cheng-Mei Tian, Yu-Qiang Nie, Jun Yao, Yu-Jie Liang, De-Feng Li, Li-Sheng Wang
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引用次数: 0

摘要

随着对胃肠道疾病(包括炎症性肠病、胃肠道癌症和胃食管反流病)发病机制认识的不断深入,人们发现了许多新的治疗靶点。这些发现为开发治疗胃肠道疾病的基因治疗策略提供了令人兴奋的可能性。这些策略包括基因替换、基因增强、基因过表达、基因功能阻断和转基因体细胞移植。在这篇综述中,我们将介绍胃肠病学领域的重要基因治疗靶点和靶向传递系统。此外,我们还全面概述了与胃肠道疾病相关的基因治疗的最新进展,并阐明了创新基因编辑技术在治疗这些疾病中的应用。这些发展正在推动胃肠道疾病治疗领域的一场革命。最后,我们讨论了当前面临的挑战(尤其是安全性、口服疗效和成本),并探讨了在胃肠道疾病临床治疗中实施基因疗法的潜在未来方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Therapeutic potential of gene therapy for gastrointestinal diseases: Advancements and future perspectives.

Therapeutic potential of gene therapy for gastrointestinal diseases: Advancements and future perspectives.

Advancements in understanding the pathogenesis mechanisms underlying gastrointestinal diseases, encompassing inflammatory bowel disease, gastrointestinal cancer, and gastroesophageal reflux disease, have led to the identification of numerous novel therapeutic targets. These discoveries have opened up exciting possibilities for developing gene therapy strategies to treat gastrointestinal diseases. These strategies include gene replacement, gene enhancement, gene overexpression, gene function blocking, and transgenic somatic cell transplantation. In this review, we introduce the important gene therapy targets and targeted delivery systems within the field of gastroenterology. Furthermore, we provide a comprehensive overview of recent progress in gene therapy related to gastrointestinal disorders and shed light on the application of innovative gene-editing technologies in treating these conditions. These developments are fueling a revolution in the management of gastrointestinal diseases. Ultimately, we discuss the current challenges (particularly regarding safety, oral efficacy, and cost) and explore potential future directions for implementing gene therapy in the clinical settings for gastrointestinal diseases.

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来源期刊
Molecular Therapy Oncolytics
Molecular Therapy Oncolytics Medicine-Oncology
CiteScore
10.90
自引率
3.50%
发文量
152
审稿时长
6 weeks
期刊介绍: Molecular Therapy — Oncolytics is an international, online-only, open access journal focusing on the development and clinical testing of viral, cellular, and other biological therapies targeting cancer.
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