UCL Huntington's Disease Centre, Department of Neurodegenerative Disease, UCL Institute of Neurology, London, WC1N 3BG, UK.
Alzheimers Res Ther. 2017 Oct 5;9(1):82. doi: 10.1186/s13195-017-0307-1.
Gene suppression approaches have emerged over the last 20 years as a novel therapeutic approach for the treatment of neurodegenerative diseases. These include RNA interference and anti-sense oligonucleotides, both of which act at the post-transcriptional level, and genome-editing techniques, which aim to repair the responsible mutant gene. All serve to inhibit the expression of disease-causing proteins, leading to the potential prevention or even reversal of the disease phenotype. In this review we summarise the main developments in gene suppression strategies, using examples from Huntington's disease and other inherited causes of neurodegeneration, and explore how these might illuminate a path to tackle other proteinopathy-associated dementias in the future.
在过去的 20 年中,基因抑制方法作为治疗神经退行性疾病的一种新的治疗方法出现了。这些方法包括 RNA 干扰和反义寡核苷酸,它们都作用于转录后水平,以及基因组编辑技术,旨在修复有问题的突变基因。所有这些方法都旨在抑制致病蛋白的表达,从而有可能预防甚至逆转疾病表型。在这篇综述中,我们使用亨廷顿病和其他遗传性神经退行性疾病的例子总结了基因抑制策略的主要进展,并探讨了它们如何为未来治疗其他与蛋白病相关的痴呆症指明道路。