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Rett Syndrome from Bench to Bedside: Recent Advances

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Journal F1000Res
Date 2018 Apr 12
PMID 29636907
Citations 8
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Abstract

Rett Syndrome is a severe neurological disorder mainly due to mutations in the methyl-CpG-binding protein 2 gene ( ). Mecp2 is known to play a role in chromatin organization and transcriptional regulation. In this review, we report the latest advances on the molecular function of Mecp2 and the new animal and cellular models developed to better study Rett syndrome. Finally, we present the latest innovative therapeutic approaches, ranging from classical pharmacology to correct symptoms to more innovative approaches intended to cure the pathology.

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