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Target intelligence / Profile preview
Methyl CpG-binding Protein 2 (MeCP2)
- Target
- MeCP2
- Molecular classification
- Transcription factor, Epigenetic regulator, DNA-binding protein
01
Overview
Methyl CpG-binding protein 2 (MeCP2) is a nuclear protein that binds specifically to methylated DNA, particularly at methylated CpG dinucleotides. It plays a critical role in the regulation of gene expression and chromatin architecture, acting as an epigenetic regulator. Mutations in MECP2 cause Rett syndrome—a severe X-linked neurodevelopmental disorder primarily affecting females. MeCP2 contains two main functional domains: Methyl-CpG Binding Domain (MBD) and Transcriptional Repression Domain (TRD).
02
Mechanism of action
MeCP2 binds to methylated DNA and recruits corepressor complexes (SIN3A/HDACs) to alter chromatin structure and regulate gene expression.
03
Biological functions
Epigenetic regulationTranscriptional repressionGene silencingRNA splicing regulationChromatin organizationNeuronal gene expression regulationSynaptic function maintenance
04
Disease associations
Rett syndromeNeurodevelopmental disordersNeurological disorders
05
Safety considerations
Aberrant MECP2 activity can lead to severe neurodevelopmental disorders like Rett syndrome.
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