Evidence map›Paper›PMID 41643021›Full record

ArticleScience (New York, N.Y.)2026

Blocking RAN translation without altering repeat RNAs rescues

Xin Jiang, Laure Schaeffer, Divya Patni, Tommaso Russo, Chao-Zong Lee, Corey Aguilar, Christine Marques, Karen Jansen-West, Marian Hruska-Plochan, Ananya Ray-Soni and 36 more

Abstract read
In one paragraph

Article in Science (New York, N.Y.), 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Review
  2. Article
  3. RAN Translation-Coupled Decay of theInternational journal of molecular sciences · 2026
    Article
  4. Review
  5. Review
  6. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

46 authors.

Xin JiangDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0003-3605-9701
Laure SchaefferUniversité de Strasbourg, Institut de Biologie Moléculaire et Cellulaire, Architecture et Réactivité de l'ARN, CNRS UPR9002, Strasbourg, France.
Divya PatniDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Tommaso RussoDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-3526-5186
Chao-Zong LeeDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Corey AguilarDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Christine MarquesDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0001-8671-0932
Karen Jansen-WestDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0002-1234-7103
Marian Hruska-PlochanDepartment of Quantitative Biomedicine, University of Zurich, Zurich, Switzerland.ORCID 0000-0002-9253-4362
Ananya Ray-SoniDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Su Min LimDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-5769-4854
Aaron HeldDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Mei YueDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.
Paula Castellanos OteroDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.
Sandeep AryalDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Hortense D A M BeaussantDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0009-0003-1014-143X
Himanish BasuDepartment of Immunology, Harvard Medical School, Boston, MA, USA.ORCID 0000-0001-8236-1157
Hiro TakakuwaWhitehead Institute for Biomedical Research, Cambridge, MA, USA.ORCID 0009-0006-6838-6407
Lillian M DaughrityDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0003-4644-7265
Nandini RameshDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0003-0860-2922
Paulo Da CostaUniversité de Strasbourg, Institut de Biologie Moléculaire et Cellulaire, Architecture et Réactivité de l'ARN, CNRS UPR9002, Strasbourg, France.ORCID 0000-0002-9749-9965
Ana Rita A A QuadrosDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-6909-2420
Matthew NolanDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-7204-5385
Charles Jourdan F ReyesDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Hayden WheelerDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Laura C MoranDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0009-0001-6940-0534
Grant GriesmanDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0009-0003-7559-259X
Benjamin WymannDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0009-0002-3270-9594
Bianca A TrombettaDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0001-7501-0666
Emma Sofia Lopez-De-SilanesDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0009-0009-8293-0634
Michael CanoriDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Gopinath KrishnanFrontotemporal Dementia Research Center, RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Yasmim Vieira Souza Da SilvaDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Gilbert ErianiUniversité de Strasbourg, Institut de Biologie Moléculaire et Cellulaire, Architecture et Réactivité de l'ARN, CNRS UPR9002, Strasbourg, France.ORCID 0000-0002-8518-4675
Mark W AlbersDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0001-7855-3455
Steven E ArnoldDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-9487-415X
Yuyu SongDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-9196-1610
Ankur JainWhitehead Institute for Biomedical Research, Cambridge, MA, USA.
Isaac M ChiuDepartment of Immunology, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-0723-4841
Yong-Jie ZhangDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0002-9900-4313
Fen-Biao GaoFrontotemporal Dementia Research Center, RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID 0000-0001-8873-5404
Brian J WaingerDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-3664-7231
Magdalini PolymenidouDepartment of Quantitative Biomedicine, University of Zurich, Zurich, Switzerland.ORCID 0000-0003-1271-9445
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0003-2959-129X
Franck MartinUniversité de Strasbourg, Institut de Biologie Moléculaire et Cellulaire, Architecture et Réactivité de l'ARN, CNRS UPR9002, Strasbourg, France.ORCID 0000-0001-9724-4025
Clotilde Lagier-TourenneDepartment of Neurology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-3058-8322

Funding

Harnessing Diverse Bioinformatic Approaches To Repurpose Drugs For Alzheimers Disease And Related DementiasR01AG058063 · NIA · MASSACHUSETTS GENERAL HOSPITAL · PI MARK W ALBERS, PETER Karl SORGER · 2018 to 2026
$7.8M
Targeting Dysregulated RNA Splicing in Neurodegenerative DiseasesRM1NS133601 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI Paul Clark Blainey, Clotilde Lagier-Tourenne · 2023 to 2026
$7.4M
Investigating Common Molecular Pathogenic Pathways in Frontotemporal Dementia Patients iPSC-Derived NeuronsR01NS057553 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Fen-Biao Gao · 2008 to 2026
$4.5M
Investigating Pathogenic Mechanisms of Frontotemporal Dementia Caused by Mutations in CHMP2B and TBK1R37NS057553 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI GAO, FEN-BIAO · 2018 to 2024
$4.3M
Understanding Frontotemporal Dementia Using Drosophila and iPSC ModelsR01NS101986 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Fen-Biao Gao · 2017 to 2026
$3.6M
Resolving the Role of Neuronal STING in Amyotrophic Lateral Sclerosis and Frontotemporal DementiaRF1NS127407 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI LAGIER-TOURENNE, CLOTILDE, WAINGER, BRIAN JASON · 2023 to 2023
$2.1M
Defining the pathogenic relationship of TDP-43 inclusions and cytoplasmic double stranded RNA in AD and FTDR01AG078377 · NIA · HARVARD MEDICAL SCHOOL · PI MARK W ALBERS · 2025 to 2026
$1.6M
NIA NIH HHS R01 AG058063NIA NIH HHS R01 AG078377NINDS NIH HHS R01 NS057553NINDS NIH HHS R01 NS101986NINDS NIH HHS R37 NS057553NINDS NIH HHS RF1 NS127407NINDS NIH HHS RM1 NS133601
6 · The paper itself

Abstract

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Indexed as

Amyotrophic Lateral SclerosisC9orf72 ProteinDNA Repeat ExpansionFrontotemporal DementiaProtein Biosynthesisran GTP-Binding ProteinRNAAnimalsCodonDipeptidesDisease Models, AnimalDNA-Binding ProteinsFemaleHEK293 CellsHumansInduced Pluripotent Stem CellsC9orf72 ProteinC9orf72 protein, humanC9orf72 protein, mouseCodonDipeptidesDNA-Binding Proteinsran GTP-Binding ProteinRNA

Identifiers

PMID41643021
PMCPMC13107528

What OpenQuestion holds

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Read underepoch 390

Registered trials

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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.