Evidence map›Paper›PMID 40697775›Full record

ArticleMolecular therapy. Methods & clinical development2025

Sustained high expression of human FVII following AAV8-mediated gene delivery in mice.

Andrea D'Amico, Bas Blits, Joëlle van den Herik, Fred de Winter, Paul H P Kaijen, Barbara Hobo, Alexander Henrik van Asbeck, Joost Verhaagen, David G M Mosmuller, Jan Voorberg

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In one paragraph

Article in Molecular therapy. Methods & clinical development, 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
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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

10 authors.

Andrea D'AmicoDepartment of Molecular Hematology, Sanquin Research and Landsteiner Laboratory of the Amsterdam UMC, 1066 CX Amsterdam, the Netherlands.
Bas BlitsSanaGen B.V., 1066 CX Amsterdam, the Netherlands.
Joëlle van den HerikLaboratory for Neuroregeneration, Netherlands Institute for Neuroscience, Royal Netherlands Academy of Sciences, 1105 BA Amsterdam, the Netherlands.
Fred de WinterLaboratory for Neuroregeneration, Netherlands Institute for Neuroscience, Royal Netherlands Academy of Sciences, 1105 BA Amsterdam, the Netherlands.
Paul H P KaijenDepartment of Molecular Hematology, Sanquin Research and Landsteiner Laboratory of the Amsterdam UMC, 1066 CX Amsterdam, the Netherlands.
Barbara HoboLaboratory for Neuroregeneration, Netherlands Institute for Neuroscience, Royal Netherlands Academy of Sciences, 1105 BA Amsterdam, the Netherlands.
Alexander Henrik van AsbeckRiboPro B.V., 5349 AB Oss, the Netherlands.
Joost VerhaagenLaboratory for Neuroregeneration, Netherlands Institute for Neuroscience, Royal Netherlands Academy of Sciences, 1105 BA Amsterdam, the Netherlands.
David G M MosmullerSanaGen B.V., 1066 CX Amsterdam, the Netherlands.
Jan VoorbergDepartment of Molecular Hematology, Sanquin Research and Landsteiner Laboratory of the Amsterdam UMC, 1066 CX Amsterdam, the Netherlands.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Factor VII (FVII) deficiency is a rare bleeding disorder with a prevalence of approximately 1:300,000-500,000 individuals. We explored whether adeno-associated virus (AAV)-mediated gene therapy can achieve durable and functional expression of human FVII (hFVII)

Indexed as

AAV5AAV8augmentation therapyFVII deficiencygene therapyliver-directed gene therapyrare bleeding disorders

Identifiers

PMID40697775
PMCPMC12282199

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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.