Department of Biochemistry, University of Colorado Boulder, Boulder, CO, USA.
Department of Molecular, Cellular and Developmental Biology, University of Colorado Boulder, Boulder, CO, USA.
Trends Mol Med. 2021 May;27(5):469-481. doi: 10.1016/j.molmed.2020.12.002. Epub 2020 Dec 29.
Skeletal muscle formation is a complex process that requires tight spatiotemporal control of key myogenic factors. Emerging evidence suggests that RNA processing is crucial for the regulation of these factors, and that multiple post-transcriptional regulatory pathways work dependently and independently of one another to enable precise control of transcripts throughout muscle development and repair. Moreover, disruption of these pathways is implicated in neuromuscular disease, and the recent development of RNA-mediated therapies shows enormous promise in the treatment of these disorders. We discuss the overlapping post-transcriptional regulatory pathways that mediate muscle development, how these pathways are disrupted in neuromuscular disorders, and advances in RNA-mediated therapies that present a novel approach to the treatment of these diseases.
骨骼肌的形成是一个复杂的过程,需要关键成肌因子的时空严格控制。新出现的证据表明,RNA 处理对于这些因子的调节至关重要,并且多个转录后调控途径相互独立和依赖地工作,以实现整个肌肉发育和修复过程中转录物的精确控制。此外,这些途径的破坏与神经肌肉疾病有关,最近 RNA 介导疗法的发展显示出在治疗这些疾病方面具有巨大的潜力。我们讨论了介导肌肉发育的重叠转录后调控途径,这些途径在神经肌肉疾病中的破坏方式,以及 RNA 介导疗法的进展,这些疗法为治疗这些疾病提供了一种新的方法。