RNA therapeutics for the treatment of blood disorders.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Anh Dao Ngo, Hoang Lam Nguyen, Safak Caglayan, Dinh-Toi Chu
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引用次数: 0

Abstract

Blood disorders are defined as diseases related to the structure, function, and formation of blood cells. These diseases lead to increased years of life loss, reduced quality of life, and increased financial burden for social security systems around the world. Common blood disorder treatments such as using chemical drugs, organ transplants, or stem cell therapy have not yet approached the best goals, and treatment costs are also very high. RNA with a research history dating back several decades has emerged as a potential method to treat hematological diseases. A number of clinical trials have been conducted to pave the way for the use of RNA molecules to cure blood disorders. This novel approach takes advantage of regulatory mechanisms and the versatility of RNA-based oligonucleotides to target genes and cellular pathways involved in the pathogenesis of specific diseases. Despite positive results, currently, there is no RNA drug to treat blood-related diseases approved or marketed. Before the clinical adoption of RNA-based therapies, challenges such as safe delivery of RNA molecules to the target site and off-target effects of injected RNA in the body need to be addressed. In brief, RNA-based therapies open novel avenues for the treatment of hematological diseases, and clinical trials for approval and practical use of RNA-targeted are crucial.

治疗血液疾病的 RNA 疗法。
血液病是指与血细胞的结构、功能和形成有关的疾病。这些疾病导致全球生命损失年数增加、生活质量下降,并加重社会保障系统的经济负担。使用化学药物、器官移植或干细胞疗法等常见的血液疾病治疗方法尚未达到最佳目标,而且治疗费用也非常高昂。已有几十年研究历史的 RNA 已成为治疗血液病的潜在方法。目前已进行了多项临床试验,为利用 RNA 分子治疗血液疾病铺平了道路。这种新方法利用基于 RNA 的寡核苷酸的调控机制和多功能性,靶向涉及特定疾病发病机制的基因和细胞通路。尽管取得了积极成果,但目前还没有治疗血液相关疾病的 RNA 药物获得批准或上市。在临床采用基于 RNA 的疗法之前,需要解决 RNA 分子安全送达靶点和注射 RNA 在体内的脱靶效应等难题。简而言之,基于 RNA 的疗法为治疗血液病开辟了新途径,而 RNA 靶向药物的临床试验审批和实际应用至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
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