Article

Brain <i>Mecp2</i> Gene Dosage and Gene Therapy Shape Multi-Omic Signatures and Putative Biomarkers in Rett Syndrome

2025-09-02

Abstract excerpt

Rett syndrome (RTT) is a neurodevelopmental disorder caused by MECP2 mutations. Like other genetic neurodevelopmental disorders, it lacks protein biomarkers to evaluate disease and therapeutic outcomes. We present a strategy to define putative biomarkers of MeCP2 dysfunction in brain with potential to delineate mechanisms and monitor therapeutic interventions. This strategy relies on a library of proteins respons...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: b81c8853-1c80-5fdc-bd37-afbc378f88e1
DOI: 10.1101/2025.08.30.673242

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Brain <i>Mecp2</i> Gene Dosage and Gene Therapy Shape Multi-Omic Signatures and Putative Biomarkers in Rett SyndromeDOI 10.1101/2025.08.30.673242Read

Meta-Analysis Identifies BDNF and Novel Common Genes Differently Altered in Cross-Species Models of Rett Syndrome.Semantic proximity Read

Identification of molecular signatures and pathways involved in Rett syndrome using a multi-omics approachSemantic proximity Read

Meta-Analysis Identifies Novel Common Genes Differently Altered in Cross-Species Models of Rett SyndromeSemantic proximity Read

RNA sequencing and proteomics approaches reveal novel deficits in the cortex of Mecp2-deficient mice, a model for Rett syndrome.Semantic proximity Read

Expanding the <i>MECP2</i> network using comparative genomics reveals potential therapeutic targets for Rett syndromeSemantic proximity Read

Convergent Cerebrospinal Fluid Proteomes and Metabolic Ontologies in Humans and Animal Models of Rett SyndromeSemantic proximity Read

Transcriptome Analysis of Brain Tissues in a MeCP2-Null Rat Model of Rett SyndromeSemantic proximity Read

Systemic Proteome Phenotypes Reveal Defective Metabolic Flexibility in Mecp2 MutantsSemantic proximity Read

Systemic proteome phenotypes reveal defective metabolic flexibility in Mecp2 mutants.Semantic proximity Read

Epigenetic Dysregulation in Rett Syndrome: Multisystem Pathophysiology, MECP2 Isoform-Specific Mechanisms, and Targeted Therapeutic StrategiesSemantic proximity Read

Epigenetic Dysregulation in Rett Syndrome: Multisystem Pathophysiology, MECP2 Isoform-Specific Mechanisms, and Targeted Therapeutic StrategiesSemantic proximity Read

MECP2pedia: A Comprehensive Transcriptome Portal for MECP2 Disease ResearchSemantic proximity Read