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首页> 外文期刊>Human gene therapy. Clinical development >Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy
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Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy

机译：在杜氏肌营养不良症中使用反义寡核苷酸的临床试验

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Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by mutations in the DMD gene, affecting 1 in 3500 newborn males. Complete loss of muscle dystrophin protein causes progressive muscle weakness and heart and respiratory failure, leading to premature death. Antisense oligonucleotides (AONs) that bind to complementary sequences of the dystrophin pre-mRNA to induce skipping of the targeted exon by modulating pre-mRNA splicing are promising therapeutic agents for DMD. Such AONs can restore the open reading frame of the DMD gene and produce internally deleted, yet partially functional dystrophin protein isoforms in skeletal muscle. Within the last few years, clinical trials using AONs have made considerable progress demonstrating the restoration of functional dystrophin protein and acceptable safety profiles following both local and systemic delivery in DMD patients. However, improvement of AON delivery and efficacy, along with the development of multiple AONs to treat as many DMD patients as possible needs to be addressed for this approach to fulfill its potential. Here, we review the recent progress made in clinical trials using AONs to treat DMD and discuss the current challenges to the development of AON-based therapy for DMD.
机译：杜兴氏肌营养不良症（DMD）是一种严重的肌肉萎缩症，由DMD基因突变引起，影响了3500名新生男性中的1个。肌营养不良蛋白的完全丧失会导致进行性肌无力以及心脏和呼吸衰竭，导致过早死亡。与肌营养不良蛋白前体mRNA的互补序列结合以通过调节前体mRNA剪接诱导靶向外显子的跳跃的反义寡核苷酸是有前途的DMD治疗剂。此类AON可以恢复DMD基因的开放阅读框，并在骨骼肌中产生内部缺失但部分功能的肌营养不良蛋白同工型。在过去的几年中，使用AON的临床试验取得了可观的进展，证明了DMD患者局部和全身分娩后功能性肌营养不良蛋白的恢复和可接受的安全性。但是，这种方法需要解决AON递送和疗效的提高，以及开发多种AON来治疗尽可能多的DMD患者的方法，以实现其潜力。在这里，我们回顾了使用AON治疗DMD的临床试验的最新进展，并讨论了基于AON的DMD治疗方法的发展面临的挑战。

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