The potential of epigenetic therapies in neurodegenerative diseases

Front Genet. 2014 Jul 14;5:220. doi: 10.3389/fgene.2014.00220. eCollection 2014.


Available treatments for neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and Huntington's disease, do not arrest disease progression but mainly help keeping patients from getting worse for a limited period of time. Increasing evidence suggests that epigenetic mechanisms such as DNA methylation and histone tail modifications are dynamically regulated in neurons and play a fundamental role in learning and memory processes. In addition, both global and gene-specific epigenetic changes and deregulated expression of the writer and eraser proteins of epigenetic marks are believed to contribute to the onset and progression of neurodegeneration. Studies in animal models of neurodegenerative diseases have highlighted the potential role of epigenetic drugs, including inhibitors of histone deacetylases and methyl donor compounds, in ameliorating the cognitive symptoms and preventing or delaying the motor symptoms of the disease, thereby opening the way for a potential application in human pathology.

Keywords: Alzheimer's disease; DNA methylation; Huntington's disease; Parkinson's disease; amyotrophic lateral sclerosis; histone deacetylase inhibitors (HDACi); histone tail modifications; therapeutic approaches.

Publication types

  • Review