Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy

Genetics and Molecular Biology

Jafar Mohseni²  Z.a.m.h. Zabidi-hussin¹  Teguh Haryo Sasongko² 

[1] ,Universiti Sains Malaysia School of Medical Sciences Human Genome CentreKubang Kerian Kelantan ,Malaysia

关键词: HDACi;    molecular therapy;    spinal muscular atrophy;   

DOI  :  10.1590/S1415-47572013000300001

来源: SciELO

【 摘 要 】

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.

【 授权许可】

CC BY   
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