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Author Lunke, Sebastian ♦ El Osta, Assam
Source SpringerLink
Content type Text
Publisher Springer US
File Format PDF
Copyright Year ©2013
Language English
Subject Domain (in DDC) Technology ♦ Medicine & health
Subject Keyword Epigenetic ♦ Chromatin ♦ Histones ♦ Spinal muscular atrophy ♦ Clinical trials ♦ Neurosciences ♦ Neurology ♦ Neurosurgery ♦ Neurobiology
Abstract Spinal muscular atrophy (SMA), a neurodegenerative disease with potentially devastating and even deadly effects on affected individuals, was first described in the late nineteenth century. Although the survival of motor neuron (SMN) gene was identified nearly 2 decades ago to be causative of the disease, neither an effective treatment nor a cure are currently available. Yet efforts are on-going to test a multitude of treatment strategies with the potential to alleviate disease symptoms in human and clinical trials. Among the most studied compounds for the treatment of SMA are histone deacetylase inhibitors. Several of these epigenetic modifiers have been shown to increase expression of the crucial SMN gene in vitro and in vivo, an effect linked to increased histone acetylation and remodeling of the chromatin landscape surrounding the SMN gene promoter. Here, we review the history and current state of use of histone deacetylase inhibitors in SMA, as well as the success of clinical trials investigating the clinical applicability of these epigenetic modifiers in SMA treatment.
ISSN 19337213
Age Range 18 to 22 years ♦ above 22 year
Educational Use Research
Education Level UG and PG
Learning Resource Type Article
Publisher Date 2013-08-31
Publisher Institution American Society for Experimental NeuroTherapeutics
Publisher Place Boston
e-ISSN 18787479
Journal NeuroRX
Volume Number 10
Issue Number 4
Page Count 11
Starting Page 677
Ending Page 687


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Source: SpringerLink