The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases.

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-04-01 Epub Date: 2024-03-12 DOI:10.1089/hum.2023.210
Alessandro Romano, Alessandra Mortellaro
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Abstract

Inflammatory diseases are conditions characterized by abnormal and often excessive immune responses, leading to tissue and organ inflammation. The complexity of these disorders arises from the intricate interplay of genetic factors and immune responses, which challenges conventional therapeutic approaches. However, the field of genetic manipulation has sparked unprecedented optimism in addressing these complex disorders. This review aims to comprehensively explore the application of gene therapy and gene editing in the context of inflammatory diseases, offering solutions that range from correcting genetic defects to precise immune modulation. These therapies have exhibited remarkable potential in ameliorating symptoms, improving quality of life, and even achieving disease remission. As we delve into recent breakthroughs and therapeutic applications, we illustrate how these advancements offer novel and transformative solutions for conditions that have traditionally eluded conventional treatments. By examining successful case studies and preclinical research, we emphasize the favorable results and substantial transformative impacts that gene-based interventions have demonstrated in patients and animal models of inflammatory diseases such as chronic granulomatous disease, cryopyrin-associated syndromes, and adenosine deaminase 2 deficiency, as well as those of multifactorial origins such as arthropathies (osteoarthritis, rheumatoid arthritis) and inflammatory bowel disease. In conclusion, gene therapy and gene editing offer transformative opportunities to address the underlying causes of inflammatory diseases, ushering in a new era of precision medicine and providing hope for personalized, targeted treatments.

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炎症性疾病中基因治疗和基因编辑的新前沿。
炎症性疾病的特点是免疫反应异常,通常是过度免疫反应,导致组织和器官发炎。这些疾病的复杂性源于遗传因素和免疫反应之间错综复杂的相互作用,这对传统的治疗方法提出了挑战。然而,基因操纵领域为解决这些复杂疾病带来了前所未有的乐观前景。本综述旨在全面探讨基因治疗和基因编辑在炎症性疾病中的应用,提供从纠正基因缺陷到精确免疫调节的各种解决方案。这些疗法在改善症状、提高生活质量,甚至实现疾病缓解方面展现出非凡的潜力。在深入探讨最近的突破和治疗应用时,我们将说明这些进展如何为传统疗法无法治愈的疾病提供新颖、变革性的解决方案。通过研究成功案例和临床前研究,我们强调了基于基因的干预措施在慢性肉芽肿病、低温蛋白相关综合征、腺苷脱氨酶 2 缺乏症等炎症性疾病患者和动物模型中,以及在关节病(骨关节炎、类风湿性关节炎)和炎症性肠病等多因素引起的炎症性疾病患者和动物模型中取得的良好效果和产生的实质性变革影响。总之,基因治疗和基因编辑为解决炎症性疾病的根本原因提供了变革性的机会,开创了精准医学的新时代,为个性化的靶向治疗带来了希望。
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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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