Evidence map›Paper›PMID 42028575›Full record

ArticleMolecular therapy. Nucleic acids2026

From N-of-1 to versatility in propionic acidemia: Antisense oligonucleotide-mediated skipping of a constitutive

Eriko Totsune, Yoichi Wada, Yasuko Mikami-Saito, Natsuko Arai-Ichinoi, Naoya Saijo, Jun Takayama, Yasuhiro Maeda, Yoko Nakajima, Osamu Ohara, Shigeo Kure and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Nucleic acids, 2026. 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

11 authors.

Eriko TotsuneDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Yoichi WadaDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Yasuko Mikami-SaitoDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Natsuko Arai-IchinoiDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Naoya SaijoDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Jun TakayamaDepartment of AI and Innovative Medicine, Tohoku University Graduate School of Medicine, Sendai 980-8575, Japan.
Yasuhiro MaedaOpen Facility Center, Fujita Health University, Toyoake 470-1192, Japan.
Yoko NakajimaDepartment of Pediatrics, Fujita Health University School of Medicine, Toyoake 470-1192, Japan.
Osamu OharaDepartment of Technology Development, Kazusa DNA Research Institute, Kisarazu 292-0818, Japan.
Shigeo KureDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.
Atsuo KikuchiDepartment of Pediatrics, Tohoku University Graduate School of Medicine, Sendai 980-8574, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Propionic acidemia is a rare autosomal recessive disorder caused by mutations in the

Indexed as

antisense oligonucleotide therapycryptic splicingMT: Oligonucleotides: Therapies and ApplicationsPCCAPCCBPCC enzyme activitypropionic acidemiapropionyl-CoA carboxylasepseudoexonsplice-switching antisense oligonucleotides

Identifiers

PMID42028575
PMCPMC13101689

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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.