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Article

Rett Syndrome: Reaching for Clinical Trials.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics - 1 Jul 2015

Pozzo-Miller Lucas, Pati Sandipan, Percy Alan K

Abstract excerpt

Rett syndrome (RTT) is a syndromic autism spectrum disorder caused by loss-of-function mutations in MECP2. The methyl CpG binding protein 2 binds methylcytosine and 5-hydroxymethycytosine at CpG sites in promoter regions of target genes, controlling their transcription by recruiting co-repressors and co-activators. Several preclinical studies in mouse models have identified rational molecular targets for drug...

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Source

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Identifiers

PMID: 25861995
DOI: 10.1007/s13311-015-0353-y
PMCID: PMC4489949

Genes

MECP2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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