ArticleScience (New York, N.Y.)2023
Mechanism of
2 registry-linked trialsOpen access · greenAbstract read
In one paragraph
Article in Science (New York, N.Y.), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 144 papers.
0numbers the graph read from it
0cells of the map it votes in
144citing papers in PubMed
49.0field-weighted citation impact, top 1% of its field
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.
NCT06782958 phase1enrolling by invitationnot on this mapstarted 2024, after this paper: background citation
A Phase I Clinical Study to Evaluate the Safety, Tolerability, and Pharmacokinetic Characteristics of FHND1002 Granules After Single and Multiple Doses in Healthy Adult Volunteers
NCT07138014 phase2not yet recruitingnot on this mapstarted 2025, after this paper: background citation
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of FHND1002 Granules in Patients With Amyotrophic Lateral Sclerosis (ALS)
Who cites it
144 citing papers in PubMed, 236 citations in OpenAlex.
- Investigational eIF2B activator DNL343 modulates the integrated stress response in preclinical models of TDP-43 pathology and individuals with ALS in a randomized clinical trial.Nature communications · 2025Trial
- The small giant actor in the pathogenesis of neurodegenerative diseases: Microtubules.Metabolic brain disease · 2026Review
- Impaired Motor Function in a Zebrafish Stathmin-2 Knockout Model.Developmental neurobiology · 2026Article
- Antisense oligonucleotide treatment following viral delivery of artificial SOD1-targeting miRNA shows improved efficacy in SOD1-G93A mice.Molecular therapy. Advances · 2026Article
- Review
- Integrative ALS therapy: gene targeted therapy by base editing and potential exercise rehabilitation as synergistic strategies for functional recovery.Physical activity and nutrition · 2026Article
- Using human 3D organoid models to gain mechanistic insight in motor neuron diseases.Nature reviews. Neuroscience · 2026Review
- Stathmin-2 mediates paracrine hormone regulation of glucagon through lysosomal trafficking in αTC1-6 cells.The Journal of physiology · 2026Article
- Prion-like Protein TDP-43: Mechanisms, Diagnosis, and Therapeutic Prospects.Pathogens (Basel, Switzerland) · 2026Review
- A mutation in the nuclear speckle and splicing factor SRRM2 is associated with multisystem proteinopathy and causes dysregulation of synapse-associated genes.RNA (New York, N.Y.) · 2026Article
- Neurabin I haploinsufficiency disrupts ion channel regulation and synaptic maturation in human cortical neurons in neurodevelopmental disorders.Molecular psychiatry · 2026Article
- Microglial TDP-43 mediates myelin refinement and represses Tyrobp cryptic exon inclusion in mice.Nature neuroscience · 2026Article
- Targeting TDP-43 in sporadic amyotrophic lateral sclerosis.Journal of neurology · 2026Review
- TDP-43 proteinopathy as a biomarker and therapeutic target in amyotrophic lateral sclerosis.Biochemical Society transactions · 2026Review
- TDP-43 promotes allergen-induced inflammation and influenza A virus replication in airway epithelium.Nature communications · 2026Article
- Pathogenicity Classification ofCells · 2026Article
- Integrated single-cell and spatial transcriptomic profiling in ALS uncovers peripheral-to-central immune infiltration and reprogramming.Nature neuroscience · 2026Article
- TDP-43 Aggregation: The Healthy-Toxic Balance of the Prion-Like Domain.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Review
- Expanding the toolbox: Emerging antisense oligonucleotide mechanisms for modulating gene expression.Molecular therapy. Nucleic acids · 2026Review
- Distinct UNC13A Haplotype Blocks Define Disease Severity and Survival in Chinese Amyotrophic Lateral Sclerosis.European journal of neurology · 2026Article
84 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
24 authors at 6 institutions in 2 countries.
Michael W BaughnLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0001-8525-8024
Ze'ev MelamedLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0002-3734-3926
Jone López-ErauskinLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.
Melinda S BeccariLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0003-1019-6388
Karen LingIonis Pharmaceuticals, Carlsbad, CA 92010, USA.ORCID 0000-0002-5895-6684
Aamir ZuberiRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.ORCID 0000-0003-0252-9760
Maximilliano PresaRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.ORCID 0000-0002-0160-6528
Elena Gonzalo-GilRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.
Roy MaimonLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.
Sonia Vazquez-SanchezLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0002-8107-5850
Som ChaturvediDepartment of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA 92093, USA.
Mariana Bravo-HernándezDepartment of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0001-8762-0357
Vanessa TaupinDepartment of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA 92093, USA.
Stephen MooreDepartment of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA 92093, USA.
Jonathan W ArtatesLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.
Eitan AcksLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.
I Sandra NdayambajeDepartment of Neurology, Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.ORCID 0000-0002-8099-2307
Ana R Agra de Almeida QuadrosDepartment of Neurology, Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.ORCID 0000-0002-6909-2420
Paayman Jafar-NejadIonis Pharmaceuticals, Carlsbad, CA 92010, USA.ORCID 0000-0001-5176-739X
Frank RigoIonis Pharmaceuticals, Carlsbad, CA 92010, USA.
C Frank BennettIonis Pharmaceuticals, Carlsbad, CA 92010, USA.ORCID 0000-0001-9887-6251
Cathleen LutzRare Disease Translational Center, The Jackson Laboratory, Bar Harbor, ME 04609, USA.
Clotilde Lagier-TourenneDepartment of Neurology, Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.ORCID 0000-0002-3058-8322
Don W ClevelandLudwig Institute for Cancer Research, University of California at San Diego, La Jolla, CA 92093, USA.ORCID 0000-0002-1934-3682
University of California San Diego · USIonis Pharmaceuticals (United States) · USJackson Laboratory · USMassachusetts General Hospital · USBroad Institute · USHebrew University of Jerusalem · IL
Funding
Shared Resource ManagementP30CA034196 · NCI · JACKSON LABORATORY · PI William Carl Skarnes · 1985 to 2026
$61.9MUCSD Shiley-Marcos Alzheimer's Disease Research Center P30P30AG062429 · NIA · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Steven Dyal Edland · 2019 to 2026
$34.9MThe Mutant Mouse Resource and Research Center at The Jackson LaboratoryU42OD010921 · OD · JACKSON LABORATORY · PI LAURA G REINHOLDT · 2012 to 2026
$21.4MNEURONAL FILAMENT SYNTHESIS, ASSEMBLY, AND FUNCTIONR01NS027036 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Bogdan Bintu, Don W Cleveland · 1989 to 2026
$11.8MTraining Program in Basic Clinical GeneticsT32GM008666 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI HAMILTON, BRUCE A · 1998 to 2021
$8.6MDetermining stathmin-2 function and potential as a therapeutic target in ALS/FTDR01NS112503 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI CLEVELAND, DON W, LAGIER-TOURENNE, CLOTILDE · 2020 to 2024
$4.1MIn vivo modelling and therapy development for stathmin-2 loss in TDP-43 proteinopathiesRF1NS124203 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI CLEVELAND, DON W, LAGIER-TOURENNE, CLOTILDE · 2021 to 2021
$2.5MMultidisciplinary training in basic and translational Alzheimer's disease researchT32AG066596 · NIA · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI BREWER, JAMES B · 2020 to 2024
$2.2MIn vivo modelling and therapy development for stathmin-2 loss in TDP-43 proteinopathiesR01NS124203 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI CLEVELAND, DON W, LAGIER-TOURENNE, CLOTILDE · 2024 to 2025
$1.6MNCI NIH HHS P30 CA034196NIA NIH HHS P30 AG062429NIA NIH HHS T32 AG066596NIGMS NIH HHS T32 GM008666NIH HHS U42 OD010921NINDS NIH HHS R01 NS027036NINDS NIH HHS R01 NS112503NINDS NIH HHS R01 NS124203NINDS NIH HHS RF1 NS124203
Abstract
Loss of nuclear TDP-43 is a hallmark of neurodegeneration in TDP-43 proteinopathies, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 mislocalization results in cryptic splicing and polyadenylation of pre-messenger RNAs (pre-mRNAs) encoding stathmin-2 (also known as SCG10), a protein that is required for axonal regeneration. We found that TDP-43 binding to a GU-rich region sterically blocked recognition of the cryptic 3' splice site in
Indexed as
DNA-Binding ProteinsGene EditingPolyadenylationRNA SplicingStathminTDP-43 ProteinopathiesAnimalsHumansMiceNeuronal OutgrowthOligonucleotides, AntisenseRNA PrecursorsRNA Splice SitesDNA-Binding ProteinsOligonucleotides, AntisenseRNA PrecursorsRNA Splice SitesStathminSTMN2 protein, humanStmn2 protein, mouseTardbp protein, mouse
Identifiers
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Read underepoch 390
Registered trials
NCT06782958ClinicalTrials.gov
NCT07138014ClinicalTrials.gov
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.