Reversal of Neurological Defects in a Mouse Model of Rett Syndrome
Top Cited Papers
- 23 February 2007
- journal article
- other
- Published by American Association for the Advancement of Science (AAAS) in Science
- Vol. 315 (5815) , 1143-1147
- https://doi.org/10.1126/science.1138389
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.Keywords
This publication has 15 references indexed in Scilit:
- Mild overexpression of MeCP2 causes a progressive neurological disorder in miceHuman Molecular Genetics, 2004
- Rett Syndrome: A Prototypical Neurodevelopmental DisorderThe Neuroscientist, 2004
- DNA methylation and Rett syndromeHuman Molecular Genetics, 2003
- A mouse Mecp2-null mutation causes neurological symptoms that mimic Rett syndromeNature Genetics, 2001
- Deficiency of methyl-CpG binding protein-2 in CNS neurons results in a Rett-like phenotype in miceNature Genetics, 2001
- A method for the generation of conditional gene repair mutations in miceNucleic Acids Research, 2001
- Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2Nature Genetics, 1999
- MeCP2 Is a Transcriptional Repressor with Abundant Binding Sites in Genomic ChromatinCell, 1997
- Selective Dendritic Alterations in the Cortex of Rett SyndromeJournal of Neuropathology and Experimental Neurology, 1995
- Purification, sequence, and cellular localization of a novel chromosomal protein that binds to Methylated DNACell, 1992