Evidence map›Paper›PMID 40487352›Full record

ArticleMolecular therapy. Nucleic acids2025

Anti-gene oligonucleotides targeting Friedreich's ataxia expanded GAA⋅TTC repeats increase Frataxin expression.

Negin Mozafari, Salomé Milagres, Tea Umek, Cristina S J Rocha, Claudia M Vargiu, Fiona Freyberger, Osama Saher, Marek Napierala, Jill S Napierala, Pontus Blomberg and 5 more

Abstract read
PubMed Full text at PMC Publisher
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. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. Article
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

15 authors.

Negin MozafariDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Salomé MilagresDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Tea UmekDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Cristina S J RochaDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Claudia M VargiuDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Fiona FreybergerDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Osama SaherDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Marek NapieralaDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Jill S NapieralaDepartment of Neurology, O'Donnell Brain Institute, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Pontus BlombergDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Per T JørgensenDepartment of Physics, Chemistry and Pharmacy, Biomolecular Nanoscale Engineering Center, University of Southern Denmark, Odense, Denmark.
Tanel PungaDepartment of Medical Biochemistry and Microbiology (IMBIM), Uppsala University, 75123 Uppsala, Sweden.
C I Edvard SmithDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.
Jesper WengelDepartment of Physics, Chemistry and Pharmacy, Biomolecular Nanoscale Engineering Center, University of Southern Denmark, Odense, Denmark.
Rula ZainDepartment of Laboratory Medicine, Karolinska Institutet, ANA Futura, Alfred Nobels Allé 8, SE-141 52 Huddinge, Stockholm, Sweden.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Friedreich's ataxia is a progressive, autosomal recessive ataxia caused, in most cases, by homozygous expansion of GAA⋅TTC triplet-repeats in the first intron of the

Indexed as

anti-geneDNA targetingfrataxinFriedreich’s ataxiaH-DNAHuntingtonś diseaseMT: Oligonucleotides: Therapies and Applicationsoligonucleotidetandem repeatstherapeutic oligonucleotide optimizationtrinucleotide repeat expansions

Identifiers

PMID40487352
PMCPMC12143624

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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.