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

Pharmacological reversal of synaptic and network pathology in human MECP2-KO neurons and cortical organoids.

EMBO molecular medicine - 11 Jan 2021

Trujillo Cleber A, Adams Jason W, Negraes Priscilla D, Carromeu Cassiano, Tejwani Leon, Acab Allan, Tsuda Ben, Thomas Charles A, Sodhi Neha, Fichter Katherine M, Romero Sarah, Zanella Fabian, Sejnowski Terrence J, Ulrich Henning, Muotri Alysson R

Abstract excerpt

Duplication or deficiency of the X-linked MECP2 gene reliably produces profound neurodevelopmental impairment. MECP2 mutations are almost universally responsible for Rett syndrome (RTT), and particular mutations and cellular mosaicism of MECP2 may underlie the spectrum of RTT symptomatic severity. No clinically approved treatments for RTT are currently available, but human pluripotent stem cell technology offers...

Read the complete abstract on PubMed

Topics

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

Share to Bridged Bicyclo Compounds, Heterocyclic
Share to Gene Knockout Techniques

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Methyl-CpG-Binding Protein 2

Share to Neurons

Share to Organoids

Share to Phenotype

Share to Pyrrolidinones

Share to Rett Syndrome

Source

Open in PubMed Open full text

Identifiers

PMID: 33501759
DOI: 10.15252/emmm.202012523
PMCID: PMC7799367

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Pharmacological reversal of synaptic and network pathology in human MECP2-KO neurons and cortical organoids.PMID 33501759Read ↗

KW-2449 and VPA exert therapeutic effects on human neurons and cerebral organoids derived from MECP2-null hESCsSemantic proximity Read ↗

Molecular and Functional Dysregulations in Novel, Rett Syndrome Patient-Derived Cerebral OrganoidsSemantic 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 ↗

Early differential impact of MeCP2 mutations on functional networks in Rett syndrome patient-derived human cerebral organoidsSemantic proximity Read ↗

MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome.Semantic proximity Read ↗

Early differential impact of MeCP2 mutations on functional networks in Rett syndrome patient-derived human cortical organoids.Semantic proximity Read ↗

Early transcriptional signatures of MeCP2 positive and negative cells in Rett syndromeSemantic proximity Read ↗

Isolation of MECP2-null Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation.Semantic proximity Read ↗

Single-nucleus profiling reveals a core disease signature and cell type-specific vulnerabilities in early Rett syndrome.Semantic proximity Read ↗

Modeling Rett Syndrome with Human Pluripotent Stem Cells: Mechanistic Outcomes and Future Clinical Perspectives.Semantic proximity Read ↗

Deciphering MECP2-associated disorders: disrupted circuits and the hope for repair.Semantic proximity Read ↗

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