Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics

G. Hu, Chen Chen
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Abstract

Review Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics Guo Hu  and Chen Chen * Division of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China. * Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422     Received: 10 October 2022 Accepted: 4 November 2022 Published: 11 January 2023   Abstract: Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.
有希望的治疗杜氏肌营养不良:利用核酸疗法恢复营养不良蛋白的表达
*华中科技大学同济医学院同济医院心脏科,心脏科遗传与分子机制湖北省重点实验室,武汉。*通信:chenchen@tjh.tjmu.edu.cn;收稿日期:2022年10月10日收稿日期:2022年11月4日发表日期:2023年1月11日摘要:杜氏肌营养不良症是由功能性肌营养不良蛋白产生不足引起的。传统的临床治疗只能轻微缓解疾病的进展,但不能完全阻止或逆转肌肉功能的下降。针对肌营养不良蛋白恢复的治疗方法目前正在开发中,其中包括外显子跳跃和停止密码子读取疗法。它们现在用于临床,而基因添加疗法处于III期临床试验。基因编辑疗法最近也首次被批准进行临床试验。本文将讨论这些新兴疗法、临床试验和未来发展方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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