Burke Medical Research Institute, 785 Mamaroneck Avenue, White Plains, 10605 NY, USA.
Expert Opin Investig Drugs. 2009 May;18(5):573-84. doi: 10.1517/13543780902810345.
Decreased histone acetyltransferase activity and transcriptional dysfunction have been implicated in almost all neurodegenerative conditions. Increasing net histone acetyltransferase activity through inhibition of the histone deacetylases (HDACs) has been shown to be an effective strategy to delay or halt progression of neurological disease in cellular and rodent models. These findings have provided firm rationale for Phase I and Phase II clinical trials of HDAC inhibitors in Huntington's disease, spinal muscular atrophy, and Freidreich's ataxia. In this review, we discuss the current findings and promise of HDAC inhibition as a strategy for treating neurological disorders. Despite the fact that HDAC inhibitors are in an advanced stage of development, we suggest other approaches to modulating HDAC function that may be less toxic and more efficacious than the canonical agents developed so far.
组蛋白乙酰转移酶活性降低和转录功能障碍与几乎所有神经退行性疾病都有关。通过抑制组蛋白去乙酰化酶 (HDAC) 增加净组蛋白乙酰转移酶活性已被证明是一种有效策略,可以延缓或阻止神经疾病在细胞和啮齿动物模型中的进展。这些发现为亨廷顿病、脊髓性肌萎缩症和弗里德赖希共济失调的 HDAC 抑制剂的 I 期和 II 期临床试验提供了坚实的依据。在这篇综述中,我们讨论了 HDAC 抑制作为治疗神经紊乱策略的现有发现和前景。尽管 HDAC 抑制剂处于开发的高级阶段,但我们建议采用其他方法来调节 HDAC 功能,这些方法可能比迄今为止开发的经典药物毒性更小、效果更好。