Promising clinical results for Huntington's disease give hope for other diseases Antisense oligonucleotides (ASOs) have the potential to reduce, restore, or modify RNA and protein expression. Thus, they can target disease pathogenesis by altering the expression of mutant proteins (1). The recent regulatory approval of ASOs for the pediatric motor neuron disease spinal muscular atrophy has provided a regulatory pathway for additional ASO therapies in other central nervous system (CNS) diseases. Developments in ASO chemistry and advances in CNS delivery methods have enabled ASOs to enter clinical trials to treat Huntington's disease (HD). There are currently no available treatments that slow or prevent progression of HD, but two ongoing ASO-based clinical programs have shown promising results. Additionally, clinical trials of ASOs to treat amyotrophic lateral sclerosis (ALS), Parkinson's disease, and Alzheimer's disease are under way, with more in development for other neurodegenerative diseases. It is hoped that ASO-based approaches will provide effective diseasemodifying therapies for HD and similar neurodegenerative diseases soon.

中文翻译：

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用于神经变性的反义寡核苷酸

亨廷顿病的有希望的临床结果为其他疾病带来希望 反义寡核苷酸 (ASO) 有可能减少、恢复或修改 RNA 和蛋白质的表达。因此，它们可以通过改变突变蛋白的表达来靶向疾病发病机制 (1)。最近对 ASO 用于小儿运动神经元疾病脊髓性肌萎缩症的监管批准为其他中枢神经系统 (CNS) 疾病的其他 ASO 疗法提供了监管途径。ASO 化学的发展和 CNS 递送方法的进步使 ASO 能够进入临床试验以治疗亨廷顿病 (HD)。目前没有可用的治疗方法来减缓或预防 HD 的进展，但两个正在进行的基于 ASO 的临床项目已经显示出有希望的结果。此外，ASOs 治疗肌萎缩侧索硬化 (ALS)、帕金森病和阿尔茨海默病的临床试验正在进行中，其他神经退行性疾病的更多研究正在进行中。希望基于 ASO 的方法能够尽快为 HD 和类似的神经退行性疾病提供有效的疾病缓解疗法。