微RNA疗法的进展:从临床前研究到临床研究。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-09-01 DOI:10.1089/hum.2024.113
Simona Brillante, Mariagrazia Volpe, Alessia Indrieri
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引用次数: 0

摘要

微小核糖核酸(miRNA)是参与各种病理生理过程的基因表达的关键调控因子。miRNAs 能够同时调节多种通路,并参与多种疾病的治疗,这使得 miRNAs 成为极具吸引力的治疗开发工具和靶点。临床前阶段的 miRNA 研究取得了长足进展,吸引了生物制药公司的大量投资。因此,越来越多基于 miRNA 的疗法已进入临床试验阶段,用于多种疾病的诊断和治疗。虽然单个 miRNA 可调控一系列广泛的 mRNA 靶点,但这也使临床试验中出现的不良反应管理变得更加复杂。一些候选药物因毒性问题而停产,这凸显了对 miRNA 疗法进行全面风险评估的必要性。尽管目前还没有基于 miRNA 的策略获得监管机构的批准,但在过去十年中,miRNAs 调控方法和纳米给药系统取得了显著进展,从而开发出了安全、耐受性良好的新型 miRNA 候选药物。在这篇综述中,我们将介绍目前处于临床前或临床阶段的 miRNA 治疗药物开发的最新进展,这些药物可用于治疗罕见遗传疾病和多因素常见疾病。我们还讨论了与 miRNA 疗法的安全性和靶向给药有关的挑战,以及如何在临床前和临床试验中确定最有效的候选疗法。
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Advances in MicroRNA Therapeutics: From Preclinical to Clinical Studies.

MicroRNAs (miRNAs) are crucial regulators of gene expression involved in various pathophysiological processes. Their ability to modulate multiple pathways simultaneously and their involvement in numerous diseases make miRNAs attractive tools and targets in therapeutic development. Significant efforts have been made to advance miRNA research in the preclinical stage, attracting considerable investment from biopharmaceutical companies. Consequently, an increasing number of miRNA-based therapies have entered clinical trials for both diagnostic and therapeutic applications across a wide range of diseases. While individual miRNAs can regulate a broad array of mRNA targets, this also complicates the management of adverse effects seen in clinical trials. Several candidates have been discontinued due to toxicity concerns, underscoring the need for comprehensive risk assessments of miRNA therapeutics. Despite no miRNA-based strategies have yet received approval from regulatory agencies, prominent progress in the miRNA modulation approaches and in the nano-delivery systems have been made in the last decade, leading to the development of novel safe and well-tolerated miRNA drug candidates. In this review, we present recent advances in the development of miRNA therapeutics currently in preclinical or clinical stages for treating both rare genetic disorders and multifactorial common conditions. We also address the challenges related to the safety and targeted delivery of miRNA therapies, as well as the identification of the most effective therapeutic candidates in preclinical and clinical trials.

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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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