Data from: Valproic acid is protective in cellular and worm models of oculopharyngeal muscular dystrophy

Objective: To explore valproic acid (VPA) as a potentially beneficial drug
in cellular and worm models of oculopharyngeal muscular dystrophy (OPMD).
Methods: Using a combination of live cell imaging and biochemical
measures, we evaluated the potential protective effect of VPA in a stable
C2C12 muscle cell model of OPMD, in lymphoblastoid cell lines derived from
patients with OPMD and in a transgenic Caenorhabditis elegans OPMD model
expressing human mutant PABPN1. Results: We demonstrated that VPA protects
against the toxicity of mutant PABPN1. Of note, we found that VPA confers
its long-term protective effects on C2C12 cell survival, proliferation,
and differentiation by increasing the acetylated level of histones.
Furthermore, VPA enhances the level of histone acetylation in
lymphoblastoid cell lines derived from patients with OPMD. Moreover,
treatment of nematodes with moderate concentrations of VPA significantly
improved the motility of the PABPN-13 Alanines worms. Conclusions: Our
results suggest that VPA helps to counteract OPMD-related phenotypes in
the cellular and C elegans disease models.