During the last decade a number of innovative treatments including gene therapies have been approved for the treatment of monogenic inherited diseases. For some neuromuscular diseases these approaches have dramatically changed the course of the disease. For others relevant challenges still remain and require disease specific approaches to overcome difficulties related to the immune response and the efficient transduction of target cells. This review provides an overview of the current development status of mutation specific treatments for neuromuscular diseases and concludes with on outlook on future developments and perspectives.

中文翻译：

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神经肌肉疾病的产后基因治疗——机遇与局限

在过去十年中，包括基因疗法在内的许多创新疗法已被批准用于治疗单基因遗传病。对于一些神经肌肉疾病，这些方法极大地改变了疾病的进程。对于其他相关挑战仍然存在，需要针对疾病的方法来克服与免疫反应和靶细胞有效转导相关的困难。本综述概述了神经肌肉疾病突变特异性治疗的当前发展状况，并总结了对未来发展和前景的展望。