Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo mutations have devasting personal, familial, and societal impacts. As the initial product of DNA transcription, RNA transcripts and their associated ribonucleoprotein complexes provide attractive targets for modulation by increasing wild-type or blocking mutant allele expression, thus relieving downstream pathological consequences. Therefore, it is unsurprising that many existing and under-development therapeutics have focused on targeting disease-associated RNA transcripts as a frontline drug strategy for these genetic disorders. This review focuses on the current range of RNA targeting modalities using examples of both dominant and recessive neurological and neuromuscular diseases.

中文翻译：

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RNA 的治疗靶向神经和神经肌肉疾病


由家族、散发或新发突变引起的神经和神经肌肉疾病对个人、家庭和社会产生毁灭性的影响。作为 DNA 转录的初始产物，RNA 转录物及其相关的核糖核蛋白复合物通过增加野生型或阻断突变等位基因表达，从而减轻下游病理后果，为调节提供有吸引力的靶标。因此，许多现有和正在开发的疗法都专注于靶向疾病相关 RNA 转录本作为这些遗传疾病的一线药物策略也就不足为奇了。本综述使用显性和隐性神经和神经肌肉疾病的例子，重点介绍当前 RNA 靶向模式的范围。