The role of histone deasetylase inhibitors on spinal muscular atrophy treatment

Spinal muscular atrophy is a neurodegenerative disease which has no effective treatment. The disease determining survival motor neuron (SMN1) gene has identical copy, SMN2. SMN1 mutations are responsible for the disease but not SMN2. SMN2 gene does not prevent the disease but attenuate disease severity. Therefore, upregulating functional SMN protein level via inducing gene expression and/or restoring splicing is an important therapeutic approach. Histone deacetylase inhibitors have been investigated for therapeutics. Investigated inhibitors for treatment of SMA are sodium butyrate, phenylbutyrate, valproic acid, TSA, SAHA, M344 and resveratrol. Although results are optimistic and clinical trials have been initiated, an effective cure has not yet been available. Thus, studies keep going to develop new effective inhibitors, which can prevent motor neuron degeneration, through structural modifications of known ones.