MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy.

MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy.

BACKGROUND:Rett Syndrome (RTT) is an Autism Spectrum Disorder and the leading cause of mental retardation in females. RTT is caused by mutations in the Methyl CpG-Binding Protein-2 (MECP2) gene and has no treatment. Our objective is to develop viral vectors for MECP2 gene transfer into Neural Stem C...

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Bibliographic Details
Main Authors:	Mojgan Rastegar, Akitsu Hotta, Peter Pasceri, Maisam Makarem, Aaron Y L Cheung, Shauna Elliott, Katya J Park, Megumi Adachi, Frederick S Jones, Ian D Clarke, Peter Dirks, James Ellis
Format:	Article
Language:	English
Published:	Public Library of Science (PLoS) 2009-08-01
Series:	PLoS ONE
Online Access:	http://europepmc.org/articles/PMC2728539?pdf=render

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