外泌体诊断和治疗杜氏肌营养不良的潜力

I. Galkin, T. Egorova
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引用次数: 0

摘要

杜氏肌营养不良是最常见的肌营养不良类型。这种病没有有效的治疗方法。最近,研究人员开始研究外泌体的治疗潜力,外泌体是细胞分泌到细胞外环境中的小泡(40-100 nm)。它们运输几种类型的大分子,包括microRNA和蛋白质,可以通过分析来评估应用治疗的效果。此外,外泌体可以用于将治疗成分(microRNA,反义寡核苷酸)递送到靶组织。下面,我们分析现有的文献,并评估使用外泌体诊断和治疗杜氏肌营养不良的可行性。我们的结论是,一旦一些技术问题得到解决,外泌体可以在研究人员和临床医生的武器库中占有一席之地。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The potential of exosomes for the diagnosis and treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is the most common type of muscular dystrophy. There is no effective cure for this disease. Recently, researchers have started to look at the therapeutic potential of exosomes — small (40–100 nm) vesicles secreted by cells into the extracellular environment. They transport a few types of macromolecules, including microRNA and proteins, that can be analyzed to estimate the efficacy of the applied therapy. Besides, exosomes can be harnessed for delivering therapeutic components (microRNA, antisense oligonucleotides) to the target tissue. Below, we analyze the available literature and assess the feasibility of using exosomes in the diagnosis and treatment of Duchenne muscular dystrophy. We conclude that exosomes can have their place in the arsenal of researchers and clinicians once some technical issues are solved.
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