Journal
SCIENCE
Volume 315, Issue 5815, Pages 1143-1147Publisher
AMER ASSOC ADVANCEMENT SCIENCE
DOI: 10.1126/science.1138389
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Funding
- Wellcome Trust [077224] Funding Source: Medline
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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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