Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Site-blocking antisense oligonucleotides as a mechanism to fine-tune MeCP2 expression.

RNA (New York, N.Y.) - 18 Nov 2024

Vanderplow Amanda M, Dodis Grace E, Rhee Yewon, Cikowski Jakub J, Gonzalez Sonia, Smith Mackenzie L, Gogliotti Rocco G

Abstract excerpt

Rett syndrome (RTT) is a neurodevelopmental disorder caused by loss-of-function mutations in the methyl-CpG-binding protein 2 (MECP2) gene. Despite its severe phenotypes, studies in mouse models suggest that restoring MeCP2 levels can reverse RTT symptomology. Nevertheless, traditional gene therapy approaches are hindered by MeCP2's narrow therapeutic window, complicating the safe delivery of viral constructs...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to 3' Untranslated Regions
Share to Brain-Derived Neurotrophic Factor

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Gene Expression Regulation

Share to Methyl-CpG-Binding Protein 2

Share to MicroRNAs

Share to Oligonucleotides, Antisense

Share to Rett Syndrome

Source

Open in PubMed Open full text

Identifiers

PMID: 39379106
DOI: 10.1261/rna.080220.124
PMCID: PMC11571808

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Site-blocking antisense oligonucleotides as a mechanism to fine-tune MeCP2 expression.PMID 39379106Read ↗

Modulating alternative splicing of MECP2 is a potential therapeutic strategy for Rett syndrome.Semantic proximity Read ↗

From Gene to Hope: Rett Syndrome and the Rise of Molecular Therapies.Semantic proximity Read ↗

State-of-the-art therapies for Rett syndrome.Semantic proximity Read ↗

Rett Syndrome: Reaching for Clinical Trials.Semantic proximity Read ↗

In Vitro Modulation of MECP2 Expression via Antisense Inhibition of miR-132-3p in SH-SY5Y CellsSemantic proximity Read ↗

Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome.Semantic proximity Read ↗

Site-directed RNA repair of endogenous Mecp2 RNA in neurons.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 ↗

Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.Semantic proximity Read ↗

Zebrafish in understanding molecular pathophysiology, disease modeling, and developing effective treatments for Rett syndrome.Semantic proximity Read ↗

Radically truncated MeCP2 rescues Rett syndrome-like neurological defects.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.