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首页> 外文期刊>Cell cycle >Spinal muscular atrophy: a new player joins the battle for SMN2 exon 7 splicing.
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Spinal muscular atrophy: a new player joins the battle for SMN2 exon 7 splicing.

机译：脊髓性肌萎缩症：新玩家加入了SMN2外显子7拼接的战斗。

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摘要

Spinal Muscular Atrophy (SMA) is a neurodegenerative disease with high impact in the human population, being the leading genetic cause of death in infancy. No cure is currently available for SMA, raising interest in the development of novel therapeutic strategies for this disease. Much of the effort in this sense has been aimed at increasing the SMN2-derived transcript levels, either by improving transcription rate or by reprogramming exon 7 splicing. Herein, we discuss recent findings on the regulation of SMN2 gene expression, focusing on splicing modulation as a therapeutic target. We review the literature regarding splicing factors involved in the regulation of exon 7 splicing in SMN2, and discuss the role played in this process by the RNA binding protein Sam68, a novel crucial regulator of SMN2 splicing.
机译：脊髓性肌萎缩症（SMA）是一种对人类有重大影响的神经退行性疾病，是婴儿期死亡的主要遗传原因。目前尚无可用于SMA的治疗方法，引起了人们对该疾病的新型治疗策略发展的兴趣。从这个意义上讲，许多努力都旨在通过提高转录速率或通过重新编程外显子7拼接来提高SMN2衍生的转录水平。在本文中，我们讨论了SMN2基因表达调控的最新发现，重点是作为治疗靶点的剪接调控。我们回顾了有关剪接因子参与SMN2的外显子7剪接调控的文献，并讨论了RNA结合蛋白Sam68（SMN2剪接的一种新的关键调控因子）在此过程中的作用。

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