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The therapeutic potential of RNA regulation in neurological disorders

机译:神经障碍RNA调控的治疗潜力

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Introduction: Gene regulation is the term used to describe the mechanisms by which a cell increases or decreases the amount of a gene product (RNA or protein). In complex organs such as the brain, gene regulation is of the utmost importance; aberrations in the regulation of specific genes can lead to neurological disorders. Understanding these mechanisms can create new strategies for targeting these disorders and progress is being made. Two drugs that function at the RNA level (nusinersen and eteplirsen) have now been approved by the FDA for the treatment of Spinomuscular atrophy and Duchenne muscular dystrophy, respectively; several other compounds for neurological disease are currently being investigated in preclinical studies and clinical trials. Areas covered: We highlight how gene regulation at the level of RNA molecules can be used as a therapeutic strategy to treat neurological disorders. We provide examples of how such an approach is being studied or used and discuss the current hurdles. Expert opinion: Targeting gene expression at the RNA level is a promising strategy to treat genetic neurological disorders. Safe administration, long-term efficacy, and potential side effects, however, still need careful evaluation before RNA therapeutics can be applied on a larger scale.
机译:简介:基因调节是用于描述细胞增加或减少基因产物(RNA或蛋白质)的量的机制的术语。在诸如大脑的复杂器官中,基因调节至关重要;在特定基因调节中的畸变可导致神经系统疾病。了解这些机制可以创造针对这些障碍的新策略,正在进行进度。两种药物在RNA水平(NUSINERSEN和ETEPLIRSEN)目前已被FDA批准,用于分别用于治疗三种血管萎缩和杜南肌营养不良症;目前正在在临床前研究和临床试验中研究几种用于神经疾病的化合物。所涵盖的区域:我们强调了RNA分子水平的基因调控如何用作治疗神经系统疾病的治疗策略。我们提供了如何研究或使用这种方法的示例,并讨论当前的障碍。专家意见:靶向RNA水平的基因表达是治疗遗传神经系统疾病的有希望的策略。然而,安全给药,长期疗效和潜在的副作用,仍然需要仔细评估RNA治疗剂在较大规模上。

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