Therapeutic Targeting of Epigenetic Components in Amyotrophic Lateral Sclerosis (ALS)
J. Lee, H. Ryu, G. Keum, Y.J. Yoon, N.W. Kowall and H. Ryu
Affiliation: VA Boston Healthcare System and Department of Neurology, Boston University School of Medicine, 150 South Huntington Avenue, Boston, MA 02130; USA.
Keywords: Amyotrophic lateral sclerosis, epigenetic components, HDAC inhibitor, motor neuron, transcription, therapeutics.
Amyotrophic lateral sclerosis (ALS) is an adult-onset motor neuron disease characterized by degeneration of
motor neuron and glial activation followed by the progressive muscle loss and paralysis. Numerous distinct therapeutic interventions
have been examined but currently ALS does not have a cure or an efficacious treatment for the disorder. Glutamate-
induced excitotoxicity, inflammation, mitochondrial dysfunction, oxidative stress, protein aggregation, transcription
deregulation, and epigenetic modifications are associated with the pathogenesis of ALS and known to be therapeutic
targets in ALS. In this review, we discuss translational pharmacological studies targeting epigenetic components to ameliorate
ALS. Understanding of the epigenetic mechanisms will provide novel insights that will further identify potential
biological markers and therapeutic approaches for treating ALS. A combination of treatments that modulate epigenetic
components and multiple targets may prove to be the most effective therapy for ALS.
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