Pallos Judit, Bodai Laszlo, Lukacsovich Tamas, Purcell Judith M, Steffan Joan S, Thompson Leslie Michels, Marsh J Lawrence
Department of Developmental and Cell Biology, University of California, Irvine, CA 92697, USA.
Hum Mol Genet. 2008 Dec 1;17(23):3767-75. doi: 10.1093/hmg/ddn273. Epub 2008 Sep 1.
Huntington's disease (HD) is associated with transcriptional dysregulation, and multiple studies with histone deacetylase (HDAC) inhibitors suggest that global approaches for restoring transcriptional balance and appropriate protein acetylation are therapeutically promising. To determine whether more targeted approaches might be effective, we have tested the impact of all the HDACs in Drosophila on Huntingtin (Htt)-induced pathology. Among the zinc-dependent or 'classic' HDACs, we find that neurodegeneration is most sensitive to levels of Rpd3. We also find that among the NAD(+)-dependent class III deacetylases, genetic or pharmacological reduction of either Sir2 or Sirt2 provides neuroprotection to Htt-challenged animals and that even greater neuroprotection is achieved when Rpd3 and Sir2 are simultaneously reduced. Our experiments suggest that longevity promoting strategies may be distinct from those that protect against neurodegeneration in Drosophila challenged with mutant human Htt. These results highlight a novel therapeutic approach for HD in the form of Sir2 inhibition and possible combinatorial inhibition of Sir2 and Rpd3.
亨廷顿舞蹈症(HD)与转录失调相关,多项针对组蛋白去乙酰化酶(HDAC)抑制剂的研究表明,恢复转录平衡和适当蛋白质乙酰化的整体方法具有治疗前景。为了确定更具针对性的方法是否有效,我们测试了果蝇中所有HDAC对亨廷顿蛋白(Htt)诱导的病理学的影响。在锌依赖性或“经典”HDAC中,我们发现神经变性对Rpd3水平最为敏感。我们还发现,在NAD(+)依赖性III类脱乙酰酶中,Sir2或Sirt2的基因或药理学降低为受Htt挑战的动物提供了神经保护作用,并且当Rpd3和Sir2同时降低时,能实现更大的神经保护作用。我们的实验表明,在受突变人类Htt挑战的果蝇中,促进长寿的策略可能与预防神经变性的策略不同。这些结果突出了一种以抑制Sir2以及可能联合抑制Sir2和Rpd3的形式用于HD的新型治疗方法。