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Review
. 2013 Sep;36(3):299-307.
doi: 10.1590/S1415-47572013000300001. Epub 2013 Aug 30.

Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy

Jafar Mohseni  1 , Z A M H Zabidi-Hussin, Teguh Haryo Sasongko
Affiliations
Review

Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy

Jafar Mohseni et al. Genet Mol Biol. 2013 Sep.

Abstract

Histone acetylation plays an important role in regulation of transcription in eukaryotic cells by promoting a more relaxed chromatin structure necessary for transcriptional activation. Histone deacetylases (HDACs) remove acetyl groups and suppress gene expression. HDAC inhibitors (HDACIs) are a group of small molecules that promote gene transcription by chromatin remodeling and have been extensively studied as potential drugs for treating of spinal muscular atrophy. Various drugs in this class have been studied with regard to their efficacy in increasing the expression of survival of motor neuron (SMN) protein. In this review, we discuss the current literature on this topic and summarize the findings of the main studies in this field.

Keywords: HDACi; molecular therapy; spinal muscular atrophy.

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References

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