Reversal of Neurological Defects in a Mouse Model of Rett Syndrome

Overview

Journal Science

Specialty Science

Date 2007 Feb 10

PMID 17289941

Citations 586

Authors

Jacky Guy

Jian Gan

Jim Selfridge

Stuart Cobb

Adrian Bird

Affiliations

Soon will be listed here.

Abstract

Rett syndrome is an autism spectrum disorder caused by mosaic expression of mutant copies of the X-linked MECP2 gene in neurons. However, neurons do not die, which suggests that this is not a neurodegenerative disorder. An important question for future therapeutic approaches to this and related disorders concerns phenotypic reversibility. Can viable but defective neurons be repaired, or is the damage done during development without normal MeCP2 irrevocable? Using a mouse model, we demonstrate robust phenotypic reversal, as activation of MeCP2 expression leads to striking loss of advanced neurological symptoms in both immature and mature adult animals.

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