ArticleEMBO molecular medicine2021
Pharmacological reversal of synaptic and network pathology in human MECP2-KO neurons and cortical organoids.
Article in EMBO molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 66 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
66 citing papers in PubMed, 1 synthesis or guideline pooled it, 103 citations in OpenAlex.
- A beginner's guide on the use of brain organoids for neuroscientists: a systematic review.Stem cell research & therapy · 2023Pooled it
- A gene therapy strategy for Deoxyhypusine Synthase (DHPS) syndrome.Research square · 2026Article
- How copy number variations shape brain developmental disorders: Unraveling the synaptic puzzle.Psychiatry and clinical neurosciences · 2026Review
- Neuromodulators Generate Multiple Context-Relevant Behaviors in Recurrent Neural Networks.Neural computation · 2026Article
- Proteomic dataset of MECP2-deficient and wild-type human brain organoids under spaceflight and ground conditions.Scientific data · 2026Article
- Human neuronal networks on micro-electrode arrays as a tool to assess genotype-phenotype correlation in CACNA1A-related disorders.Stem cell reports · 2026Article
- Pluripotent stem cells-based neural organoids for modelling human brain development and diseases.Cell & bioscience · 2025Review
- MeCP2 regulates telencephalic development in human cerebral organoids.Cell reports · 2025Article
- Mecp2 deficiency impairs microscale cortical network topology and dynamics in a Rett syndrome mouse model.bioRxiv : the preprint server for biology · 2025Article
- WhenAPL bioengineering · 2025Review
- CYFIP1 governs the development of cortical axons by modulating calcium availability.Nature communications · 2025Article
- Genetic Manipulation in Organoid Models and Their Applications in Central Nervous System Pathologies.Molecular neurobiology · 2025Review
- Impact of intermittent lead exposure on hominid brain evolution.Science advances · 2025Article
- Article
- Zebrafish mecp2 null-mutation increases anxiety and cortisol levels but no change in adult social preference and larval chemically-induced hyperlocomotion.BMC neuroscience · 2025Article
- Exploring the complexity of MECP2 function in Rett syndrome.Nature reviews. Neuroscience · 2025Review
- Review
- Differentiation Defect Into GABAergic Neurons in Cerebral Organoids From Autism Patients.CNS neuroscience & therapeutics · 2025Article
- Modelling human brain development and disease with organoids.Nature reviews. Molecular cell biology · 2025Review
- A Blueprint for Translational Precision Medicine in Autism Spectrum Disorder and Related Neurogenetic Syndromes.Journal of child and adolescent psychopharmacology · 2025Review
6 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 3 institutions in 2 countries.
Funding
Abstract
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 a platform to identify neuropathology and test candidate therapeutics. Using a strategic series of increasingly complex human stem cell-derived technologies, including human neurons, MECP2-mosaic neurospheres to model RTT female brain mosaicism, and cortical organoids, we identified synaptic dysregulation downstream from knockout of MECP2 and screened select pharmacological compounds for their ability to treat this dysfunction. Two lead compounds, Nefiracetam and PHA 543613, specifically reversed MECP2-knockout cytologic neuropathology. The capacity of these compounds to reverse neuropathologic phenotypes and networks in human models supports clinical studies for neurodevelopmental disorders in which MeCP2 deficiency is the predominant etiology.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.