Neurological disorders are a diverse group of conditions that pose an increasing health burden worldwide. There is a general lack of effective therapies due to multiple reasons, of which a key obstacle is the presence of the blood–brain barrier, which limits drug delivery to the central nervous system, and generally restricts the pool of candidate drugs to small, lipophilic molecules. However, in many cases, these are unable to target key pathways in the pathogenesis of neurological disorders.

中文翻译：

---

CNS 疾病的 RNA 疗法

神经系统疾病是一组不同的疾病，在全世界范围内造成日益严重的健康负担。由于多种原因，普遍缺乏有效的治疗方法，其中一个关键障碍是血脑屏障的存在，它限制了药物向中枢神经系统的输送，并且通常将候选药物限制在小的、亲脂性的药物范围内。分子。然而，在许多情况下，这些药物无法针对神经系统疾病发病机制的关键途径。