Evidence map›Paper›PMID 41744818›Full record

ReviewCells2026

Gene Therapy Advancements in Age-Related Macular Degeneration Treatment.

Efstratia Amaxilati, Eleftherios Chatzimichail, Georgios N Tsiropoulos, Lorenzo Motta, Theo Empeslidis, Zisis Gatzioufas, Georgios D Panos

Abstract readReview
In one paragraph

Review in Cells, 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

7 authors.

Efstratia AmaxilatiDepartment of Ophthalmology, AHEPA University Hospital, School of Medicine, Aristotle University of Thessaloniki, 54636 Thessaloniki, Greece.ORCID 0009-0009-7071-1557
Eleftherios ChatzimichailDepartment of Ophthalmology, University Hospital of Basel, 4056 Basel, Switzerland.ORCID 0000-0003-4283-5260
Georgios N TsiropoulosDepartment of Ophthalmology, AHEPA University Hospital, School of Medicine, Aristotle University of Thessaloniki, 54636 Thessaloniki, Greece.ORCID 0000-0002-2441-545X
Lorenzo MottaEye Unit, Department of Neuroscience, University of Padua, 35131 Padua, Italy.
Theo EmpeslidisVantage Biosciences, London SW1Y 5ES, UK.ORCID 0009-0000-1545-6199
Zisis GatzioufasDepartment of Ophthalmology, University Hospital of Basel, 4056 Basel, Switzerland.
Georgios D PanosDepartment of Ophthalmology, AHEPA University Hospital, School of Medicine, Aristotle University of Thessaloniki, 54636 Thessaloniki, Greece.ORCID 0000-0001-8399-7456

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Age-related macular degeneration (AΜD) remains a leading cause of irreversible vision loss. Ιn neovascular AΜD (nAΜD), frequent intravitreal anti-VΕGF injections create substantial treatment burden, while approved therapies for geographic atrophy (GA) provide modest slowing of progression. Ocular gene therapy aims to achieve sustained intraocular expression of therapeutic proteins after a single administration. Τhis review summarises the biological rationale, vector platforms, and delivery routes relevant to AΜD, with emphasis on adeno-associated virus (AAV) systems, capsid engineering, and compartment-specific administration (intravitreal, subretinal, and suprachoroidal). We synthesise the clinical landscape for sustained anti-VΕGF expression approaches in nAΜD and complement-modulating strategies for GA, and highlight how trials increasingly prioritise injection-burden reduction, anatomical endpoints, and biomarkers of target engagement. Κey challenges include intraocular inflammation and neutralising antibodies (particularly with intravitreal dosing), variability and durability of transgene expression, surgical risks associated with subretinal delivery, and practical constraints related to manufacturing scale, cost, and long-term safety surveillance for non-removable therapies. Overall, gene therapy offers a plausible route towards durable, mechanism-targeted AΜD management, but its clinical role will depend on robust controlled trials and multi-year follow-up.

Indexed as

Genetic TherapyMacular DegenerationAnimalsDependovirusGene Therapy AgentsGenetic VectorsHumansadeno-associated virusage-related macular degenerationcomplementgene therapygeographic atrophyintravitrealneovascular AMDsubretinalsuprachoroidalVΕGF

Identifiers

PMID41744818
PMCPMC12938930

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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.