Evidence map›Paper›PMID 41394711›Full record

ArticlebioRxiv : the preprint server for biology2025

U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis.

Puja R Mehta, Tomas Solomon, Sarah Pickles, Peter Harley, Michela Barioglio, Christoph Schweingruber, Alessandro Marrero-Gagliardi, Yujing Gao, Francesca Mattedi, Simone Barattucci and 11 more

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors.

Puja R MehtaDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0002-0255-407X
Tomas SolomonUK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.ORCID 0000-0003-2600-6948
Sarah PicklesDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0001-7956-4586
Peter HarleyDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0001-6689-0227
Michela BarioglioDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0009-0009-8360-2536
Christoph SchweingruberUK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.ORCID 0000-0003-4505-9068
Alessandro Marrero-GagliardiDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0002-8470-4085
Yujing GaoUK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.ORCID 0000-0002-4542-7062
Francesca MattediDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0002-0306-5796
Simone BarattucciDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0009-0006-8380-2931
Lilian Tsai-Wei LinDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0003-2769-8763
Eugeni RyadnovDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0009-0003-9921-1755
Matteo ZanovelloDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0003-3343-1547
Alexander J CammackDepartment of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.ORCID 0000-0002-9624-9067
Adrian M IsaacsDepartment of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, London, UK.ORCID 0000-0002-6820-5534
Juan BurroneCentre for Developmental Neurobiology and MRC Centre for Neurodevelopmental Disorders, King's College London, London, UK.ORCID 0000-0003-1233-668X
Christopher E ShawUK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.ORCID 0000-0003-0251-0146
Matthew J KeussDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0002-2628-4115
Leonard PetrucelliDepartment of Neuroscience, Mayo Clinic, Jacksonville, FL, USA.ORCID 0000-0003-2959-129X
Pietro FrattaDepartment of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, UCL Queen Square Motor Neuron Disease Centre, London, UK.ORCID 0000-0002-8762-8188
Marc-David RueppUK Dementia Research Institute, Institute of Psychiatry, Psychology & Neuroscience, King's College London, London, UK.ORCID 0000-0003-3264-9800

Funding

Human Biomarkers CoreU54NS123743 · NINDS · STANFORD UNIVERSITY · PI GITLER, AARON D. · 2021 to 2025
$8.2M
NINDS NIH HHS U54 NS123743Wellcome Trust
6 · The paper itself

Abstract

TDP-43 nuclear depletion in amyotrophic lateral sclerosis (ALS) causes de-repression of cryptic exons (CEs) in multiple transcripts, including

Identifiers

PMID41394711
PMCPMC12697360

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.