ArticleNature2025
CRISPR activation for SCN2A-related neurodevelopmental disorders.
Erratum issuedAbstract read
In one paragraph
Article in Nature, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 14 papers.
0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed.
- The Genetic Architecture of Chronic Cough: From Sensory Hypersensitivity to Treatable Trait.Pulmonary therapy · 2026Review
- Organoid intelligence: a promising paradigm for autism spectrum disorder research.Molecular psychiatry · 2026Review
- Mild malformation of cortical development with oligodendroglial hyperplasia in epilepsy (MOGHE): genetics, mechanisms and precision therapy.Acta neuropathologica · 2026Review
- Next generation technologies for CRISPR-based epigenome and transcriptional modulation.Current opinion in biomedical engineering · 2026Article
- Mechanisms of SCN2A loss of function do not predict presence or phenotype of epilepsy.Epilepsia · 2026Article
- Review
- A hit for base editing: treatment of developmental epilepsy in a mouse model.The Journal of clinical investigation · 2026Article
- Degraded sensory coding in a mouse model ofbioRxiv : the preprint server for biology · 2025Article
- CRISPR activation tackles neurodevelopmental disorders.Nature reviews. Drug discovery · 2025Article
- Effect of Alzheimer's disease medications on neurocognitive outcomes in children and adolescents with autism spectrum disorder and low IQ: a scoping review.Translational psychiatry · 2025Article
- Large-scale discovery of neural enhancers for cis-regulation therapies.bioRxiv : the preprint server for biology · 2025Article
- Transgenic mouse models of sodium and potassium channelopathies in epilepsy: insights into disease mechanisms and therapeutics.Bioscience reports · 2025Review
- Tunable, proteolytic dosage control of CRISPR-Cas systems enables precise gene therapy for dosage sensitive disorders.bioRxiv : the preprint server for biology · 2025Article
- Novel advanced patient-derivedFrontiers in neurologyReview
Corrections and comments
- Erratum issued
Authors and funding
28 authors.
Serena Tamura *Department of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Andrew D Nelson *Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Perry W E Spratt *Weill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Elizabeth C HamadaWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0009-0008-0614-8141
Xujia ZhouDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Henry KyoungWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Zizheng LiDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Coline ArnouldDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Vladyslav BarskyiDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Beniamin KrupkinDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0000-0002-7457-7342
Kiana YoungDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Jingjing ZhaoDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Stephanie S HoldenDepartment of Neurology, University of California San Francisco, San Francisco, CA, USA.
Atehsa SahagunWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0000-0001-7521-3279
Caroline M KeeshenWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Congyi LuStanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.
Roy Ben-ShalomWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Sunrae E TalomaWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0000-0002-7210-8614
Selin SchamilogluWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.
Ying C LiWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0009-0009-4792-8336
Lia MinDepartment of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA.ORCID 0000-0002-3977-1684
Paul M JenkinsDepartment of Pharmacology, University of Michigan Medical School, Ann Arbor, MI, USA.ORCID 0000-0002-4207-5823
Jen Q PanStanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.
Jeanne T PazDepartment of Neurology, University of California San Francisco, San Francisco, CA, USA.ORCID 0000-0001-6339-8130
Stephan J SandersWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA.ORCID 0000-0001-9112-5148
Navneet MatharuDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA.
Nadav AhituvDepartment of Bioengineering and Therapeutic Sciences, University of California San Francisco, San Francisco, CA, USA. nadav.ahituv@ucsf.edu.ORCID 0000-0002-7434-8144
Kevin J BenderWeill Institute for Neurosciences, University of California San Francisco, San Francisco, CA, USA. kevin.bender@ucsf.edu.ORCID 0000-0001-7084-1532
Funding
Research BaseP30DK063720 · NIDDK · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI GERMAN, MICHAEL S · 2003 to 2019
$21.8MProject 3 - Development and investigation of murine models of channelopathy-associated epilepsyU54NS108874 · NINDS · NORTHWESTERN UNIVERSITY AT CHICAGO · PI KEARNEY, JENNIFER A · 2018 to 2022
$12.1MPREDOCTORAL TRAINING IN NEUROBIOLOGYT32GM007449 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI KREITZER, ANATOL · 1985 to 2017
$7.4MConvergent mechanisms for neurodevelopmental disorder genesR01MH126960 · NIMH · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI Nadav Ahituv, Kevin J Bender · 2021 to 2026
$3.5MPredoctoral Training in NeurobiologyT32NS115706 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SAMUEL JEREMY PLEASURE · 2020 to 2026
$3.4MExperience-Dependent Reorganization of Excitatory Synapse ConnectivityR01MH118298 · NIMH · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI PAN, JEN QIAN, POPESCU, GABRIELA K · 2019 to 2023
$3.3MHigh-Throughput Screens for CaV3.3 Selective T-type Calcium Channel ModulatorsR01MH115045 · NIMH · BROAD INSTITUTE, INC. · PI COTTRELL, JEFFREY RICHARD, PAN, JEN QIAN · 2018 to 2020
$2.3MRole of Myeloid And CD4+ T Immune Cells in Post-Traumatic PlasticityR01NS121287 · NINDS · J. DAVID GLADSTONE INSTITUTES · PI Jeanne T Paz · 2022 to 2026
$2.2MCRISPRa-based rescue of sensorimotor deficits in the Scn2a+/- mouse model of autism spectrum disorderR01MH136475 · NIMH · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Kevin J Bender, Daniel Feldman · 2024 to 2026
$2.0MThe function of SCN2A in neocortexR01MH125978 · NIMH · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BENDER, KEVIN J · 2021 to 2025
$2.0MRole of Thalamus in Post-stroke epileptogenesisR00NS078118 · NINDS · J. DAVID GLADSTONE INSTITUTES · PI PAZ, JEANNE T · 2014 to 2016
$747kBD FACSAria Fusion Cell SorterS10OD021822 · OD · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI LEE, MICHAEL R. · 2016 to 2016
$573kNIDDK NIH HHS P30 DK063720NIGMS NIH HHS T32 GM007449NIH HHS S10 OD021822NIMH NIH HHS F32 MH125536NIMH NIH HHS K99 MH135209NIMH NIH HHS R01 MH115045NIMH NIH HHS R01 MH118298NIMH NIH HHS R01 MH125978NIMH NIH HHS R01 MH126960NIMH NIH HHS R01 MH136475NINDS NIH HHS K99 NS078118NINDS NIH HHS R00 NS078118NINDS NIH HHS R01 NS121287NINDS NIH HHS T32 NS115706NINDS NIH HHS U54 NS108874
Abstract
Most neurodevelopmental disorders with single gene diagnoses act via haploinsufficiency, in which only one of the two gene copies is functional
Indexed as
Clustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsGenetic TherapyNAV1.2 Voltage-Gated Sodium ChannelNeurodevelopmental DisordersAnimalsDependovirusDisease Models, AnimalFemaleGene Knock-In TechniquesHaploinsufficiencyHumansMaleMicePhenotypeProof of Concept StudyNAV1.2 Voltage-Gated Sodium ChannelSCN2A protein, humanScn2a protein, mouse
Identifiers
What OpenQuestion holds
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Read underepoch 390
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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.