Evidence map›Paper›PMID 41404501›Full record

ArticleMolecular therapy. Nucleic acids2025

Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoids.

Amy Leung, Pedro R L Perdigão, Almudena Sacristan-Reviriego, Paul E Sladen, Erika A Aguzzi, Farah O Rezek, Kalliopi Ziaka, Rosellina Guarascio, Kwan-Leong Hau, Michael E Cheetham and 1 more

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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. 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

11 authors.

Amy LeungUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Pedro R L PerdigãoUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Almudena Sacristan-ReviriegoUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Paul E SladenUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Erika A AguzziUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Farah O RezekUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Kalliopi ZiakaUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Rosellina GuarascioUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Kwan-Leong HauUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Michael E CheethamUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
Jacqueline van der SpuyUniversity College London Institute of Ophthalmology, University College London, London EC1V 9EL, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Inherited retinal dystrophies (IRDs) are a group of incurable, genetically heterogeneous diseases that cause progressive degeneration of the retina, leading to the loss of vision. Genome editing technologies offer a powerful prospect for mutation correction and single-dose cures for these diseases. Here, we investigated the potential of adenine base editing (ABE) to correct a panel of causative genetic variations in patient-derived induced pluripotent stem cells (iPSCs) and identified parameters that can efficiently correct a pathogenic variation in the

Indexed as

base editingCas9CRISPRinduced pluripotent stem cellinherited retinal dystrophyLeber congenital amaurosisMT: RNA/DNA editingretinal organoid

Identifiers

PMID41404501
PMCPMC12704276

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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.