Rett syndrome from bench to bedside: recent advances.

Authors :: Ehinger Y
Matagne V
Villard L
Roux JC
Source :: F1000Research [F1000Res] 2018 Mar 26; Vol. 7, pp. 398. Date of Electronic Publication: 2018 Mar 26 (Print Publication: 2018).
Publication Year :: 2018
Abstract: Rett Syndrome is a severe neurological disorder mainly due to de novo mutations in the methyl-CpG-binding protein 2 gene ( MECP2 ). 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.<br />Competing Interests: No competing interests were disclosed.No competing interests were disclosed.No competing interests were disclosed.