Evidence map›Paper›PMID 40927767›Full record

ArticleMolecular therapy. Methods & clinical development2025

iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy.

Estelle Berreur, Giacomo Lazzaroni, Cyrill Roth, Marco Zihlmann, Martina Stirn, Ramona Matheis, Rebecca Xicluna, Ekaterina Breous-Nystrom, Adrian B Roth

Abstract read
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. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Review
  2. Article
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  6. Review
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.

Estelle BerreurRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Giacomo LazzaroniRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Cyrill RothInstitute of Human Biology, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Marco ZihlmannRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Martina StirnRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Ramona MatheisRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Rebecca XiclunaRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Ekaterina Breous-NystromRoche Pharma Research and Early Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.
Adrian B RothPrecision Safety, Pharma Product Development, Roche Innovation Center Basel, CH-4070 Basel, Switzerland.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors are widely used in gene therapy, particularly for liver-targeted treatments. However, predicting human-specific outcomes, such as transduction efficiency and hepatotoxicity, remains challenging. Reliable

Indexed as

AAV serotypeAAV transductionAAV vectorsgene therapyHepaRGHepatocyteHepG2in vitro modelsiPSCliverorganoids

Identifiers

PMID40927767
PMCPMC12415976

What OpenQuestion holds

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Registered trials

None linked

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.