Evidence map›Paper›PMID 40825661›Full record

Trial reportThe British journal of ophthalmology2026

Safety and vision outcomes of subretinal gene supplementation therapy in

Felix F Reichel, M Dominik Fischer, Katarina Stingl, Laura Kuehlewein, Immanuel Seitz, Tobias Peters, Melanie Ziegler, Barbara Wilhelm, Susanne Kohl, Nicole Weisschuh and 12 more

Abstract readClinical Trial, Phase IIClinical Trial, Phase I
In one paragraph

Trial report in The British journal of ophthalmology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. 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

22 authors.

Felix F Reichel *University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.ORCID http://orcid.org/0000-0001-9325-5616
M Dominik Fischer *University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Katarina Stingl *University Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.ORCID http://orcid.org/0000-0002-8132-911X
Laura KuehleweinUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.ORCID http://orcid.org/0000-0003-1906-8681
Immanuel SeitzUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.ORCID http://orcid.org/0000-0002-3086-6425
Tobias PetersUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Melanie ZieglerUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Barbara WilhelmUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Susanne KohlMolecular Genetics Laboratory, Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Nicole WeisschuhInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany.
Peter MartusInstitute of Clinical Epidemiology and Applied Biometrics, University of Tübingen, Tübingen, Germany.
Mathias SeeligerInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany.
Regine MuehlfriedelInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany.
Francois Paquet-DurandInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany.
Stephen H TsangDepartment of Ophthalmology, Columbia University, New York, NY, USA.
Karl Ulrich Bartz-SchmidtUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Marius UeffingInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany.
Eberhart ZrennerUniversity Eye Hospital, Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Germany.
Martin BielDepartment of Pharmacy, Center for Drug Research, LMU Munich, Munich, Germany stylianos.michalakis@cup.uni-muenchen.de martin.biel@cup.uni-muenchen.de bernd.wissinger@med.uni-tuebingen.de.
Bernd WissingerInstitute for Ophthalmic Research, Centre for Ophthalmology, Centre for Ophthalmology, University Hospital Tubingen, Tübingen, Germany stylianos.michalakis@cup.uni-muenchen.de martin.biel@cup.uni-muenchen.de bernd.wissinger@med.uni-tuebingen.de.
Stylianos MichalakisDepartment of Ophthalmology, LMU University Hospital, LMU Munich, Munich, Germany stylianos.michalakis@cup.uni-muenchen.de martin.biel@cup.uni-muenchen.de bernd.wissinger@med.uni-tuebingen.de.ORCID http://orcid.org/0000-0001-5092-9238
RD-Cure Consortium

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purpose

methodsIn an open-label, non-randomised controlled phase I/IIa trial, nine patients with biallelic

resultsThe mean patient age was 40.1 years, with baseline BCVA ranging from 40 to 82 letters (0.9-0.1 logMAR). No systemic adverse events occurred, and most ocular events resolved without treatment. Persistent adverse events included small peripheral atrophic areas (n=2), disturbed colour discrimination (n=3), cataract (n=1), slight central retinal thinning (n=5) and moderate visual acuity loss (n=2, 1 in each dose group). BCVA, full-field stimulus thresholds and other visual function measures showed statistically non-significant changes, with a trend towards worsening of retinal sensitivity in the treated eyes.

conclusionSubretinal gene therapy with AAV8.hPDE6A did not improve visual function over 1 year and posed risks, including central retinal thinning and visual acuity decline. This is in contrast to the safety and efficacy profile established in preclinical models.

Indexed as

Cyclic Nucleotide Phosphodiesterases, Type 6Genetic TherapyRetinitis PigmentosaVisual AcuityAdultContrast SensitivityDark AdaptationDependovirusEye ProteinsFemaleGenetic VectorsHumansMaleMiddle AgedTomography, Optical CoherenceTreatment OutcomeCyclic Nucleotide Phosphodiesterases, Type 6Eye ProteinsPDE6A protein, humanClinical TrialDegenerationDystrophyRetina

Identifiers

PMID40825661
PMCPMC12911619

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.