Introduction Ribonucleic acid (RNA) therapeutics are a new class of drugs whose importance is highlighted by the growing number of molecules in the clinic. Sources of data We focus on RNA therapeutics for neurogenetic disorders, which are broadly defined as diseases with a genetic background and with at least one clinical sign affecting the nervous system. A systematic search identified 14 RNA drugs approved by FDA and many others in development. Areas of agreement The field of RNA therapeutics is changing the therapeutic scenario across many disorders. Areas of controversy Despite its recent successes, RNA therapeutics encountered several hurdles and some clinical failures. Delivery to the brain represents the biggest challenge. Growing points The many advantages of RNA drugs make the development of these technologies a worthwhile investment. Areas timely for developing research Clinical failures stress the importance of implementing clinical trial design and optimizing RNA molecules to hold the promise of revolutionizing the treatment of human diseases.

中文翻译：

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RNA疗法治疗神经系统疾病

简介 核糖核酸 (RNA) 疗法是一类新型药物，临床中分子数量的不断增加凸显了其重要性。数据来源 我们专注于神经遗传性疾病的 RNA 疗法，神经遗传性疾病被广泛定义为具有遗传背景且至少具有一种影响神经系统的临床症状的疾病。系统性检索确定了 FDA 批准的 14 种 RNA 药物以及许多其他正在开发的药物。共识领域 RNA 治疗领域正在改变许多疾病的治疗方案。争议领域 尽管 RNA 疗法最近取得了成功，但它遇到了一些障碍和一些临床失败。向大脑的传递是最大的挑战。增长点 RNA药物的诸多优点使得这些技术的开发成为一项值得投资。适时开展研究的领域 临床失败强调了实施临床试验设计和优化 RNA 分子的重要性，以实现人类疾病治疗的革命性变革。