Evidence map›Paper›PMID 42436859›Full record

ArticleMolecular therapy. Advances2026

Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency.

Anna Keegan, Özge Çetin, Ellie M Chilcott, Juan Antinao Diaz, Simon Eaton, Simon N Waddington, John R Counsell, Shamima Rahman, Rajvinder Karda

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Advances, 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

9 authors.

Anna KeeganEGA-Institute for Women's Health, University College London, London, UK.
Özge ÇetinEGA-Institute for Women's Health, University College London, London, UK.
Ellie M ChilcottEGA-Institute for Women's Health, University College London, London, UK.
Juan Antinao DiazEGA-Institute for Women's Health, University College London, London, UK.
Simon EatonUnit of Paediatric Surgery, UCL Institute of Child Health, London, UK.
Simon N WaddingtonEGA-Institute for Women's Health, University College London, London, UK.
John R CounsellResearch Department of Targeted Intervention, UCL Division of Surgery and Interventional Science, Charles Bell House, London, UK.
Shamima RahmanMitochondrial Research Group, Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health, and Metabolic Unit, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Rajvinder KardaEGA-Institute for Women's Health, University College London, London, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pyruvate dehydrogenase complex deficiency (PDHD) is a severe mitochondrial disorder most frequently caused by pathogenic variants in

Indexed as

AAV-Fadeno-associated virusclincal translation and pyruvate dehydrogenase deficiencygene supplementationneonatal gene therapypreclinical

Identifiers

PMID42436859
PMCPMC13355638

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.