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miRNAs 与肌肉再生:杜氏肌营养不良症的治疗靶点。

MiRNAs and Muscle Regeneration: Therapeutic Targets in Duchenne Muscular Dystrophy.

机构信息

Department of Experimental Biology, Faculty of Experimental Sciences, University of Jaen, Paraje Las Lagunillas s/n, 23009 Jaen, Spain.

Medina Foundation, Technology Park of Health Sciences, Av. del Conocimiento 34, 18016 Granada, Spain.

出版信息

Int J Mol Sci. 2021 Apr 19;22(8):4236. doi: 10.3390/ijms22084236.

Abstract

() are small non-coding required for the post-transcriptional control of gene expression. MicroRNAs play a critical role in modulating muscle regeneration and stem cell behavior. Muscle regeneration is affected in muscular dystrophies, and a critical point for the development of effective strategies for treating muscle disorders is optimizing approaches to target muscle stem cells in order to increase the ability to regenerate lost tissue. Within this framework, miRNAs are emerging as implicated in muscle stem cell response in neuromuscular disorders and new methodologies to regulate the expression of key microRNAs are coming up. In this review, we summarize recent advances highlighting the potential of miRNAs to be used in conjunction with gene replacement therapies, in order to improve muscle regeneration in the context of Duchenne Muscular Dystrophy (DMD).

摘要

()是转录后基因表达调控所必需的小型非编码 RNA。microRNAs 在调节肌肉再生和干细胞行为方面发挥着关键作用。肌肉再生在肌肉疾病中受到影响,开发治疗肌肉疾病的有效策略的关键点是优化针对肌肉干细胞的方法,以提高再生丢失组织的能力。在这个框架内,microRNAs 被认为与神经肌肉疾病中的肌肉干细胞反应有关,并且出现了新的方法来调节关键 microRNAs 的表达。在这篇综述中,我们总结了最近的进展,强调了 microRNAs 与基因替代疗法结合使用的潜力,以改善杜氏肌营养不良症(DMD)背景下的肌肉再生。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fd1e/8072594/4069525e7a5b/ijms-22-04236-g001.jpg

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