Evidence map›Paper›PMID 40171277›Full record

ArticleMolecular therapy. Nucleic acids2025

CAG-targeted brain-permeable therapy tested in biallelic humanized polyQ mouse models.

Magdalena Surdyka, Żaneta Kalinowska-Pośka, Anna Niewiadomska-Cimicka, Ewelina Jesion, Agnieszka Fiszer, Elisabeth Singer-Mikosch, Lorraine Fievet, Lukasz Przybyl, Nicholas S Caron, Michael R Hayden and 3 more

Abstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 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

13 authors.

Magdalena SurdykaInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.
Żaneta Kalinowska-PośkaInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.
Anna Niewiadomska-CimickaInstitute of Genetics and Molecular and Cellular Biology (IGBMC), INSERM U1258, CNRS UMR7104, University of Strasbourg, Illkirch, France.
Ewelina JesionInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.
Agnieszka FiszerInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.
Elisabeth Singer-MikoschDepartment of Human Genetics, Ruhr University Bochum, Bochum, Germany.
Lorraine FievetInstitute of Genetics and Molecular and Cellular Biology (IGBMC), INSERM U1258, CNRS UMR7104, University of Strasbourg, Illkirch, France.
Lukasz PrzybylInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.
Nicholas S CaronCentre for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada.
Michael R HaydenCentre for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, Department of Medical Genetics, University of British Columbia, Vancouver, BC, Canada.
Huu Phuc NguyenDepartment of Human Genetics, Ruhr University Bochum, Bochum, Germany.
Yvon TrottierInstitute of Genetics and Molecular and Cellular Biology (IGBMC), INSERM U1258, CNRS UMR7104, University of Strasbourg, Illkirch, France.
Maciej FigielInstitute of Bioorganic Chemistry, Polish Academy of Sciences, Poznań, Poland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

In polyglutamine (polyQ) diseases, including Huntington disease (HD) and spinocerebellar ataxia type 3 (SCA3), targeting the mutant CAG tract in mRNA could be a therapeutic strategy for lowering pathogenic protein. We explored the viability of this therapeutic strategy

Indexed as

AAV-PHP.eBblood-brain barrierCAG repeats targetinggene therapyHuntington diseaseMT: Oligonucleotides: Therapies and Applicationsneurodegenerative diseaseSCA3short hairpin RNAshRNAspinocerebellar ataxia type 3systemic delivery

Identifiers

PMID40171277
PMCPMC11960632

What OpenQuestion holds

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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.