Evidence map›Paper›PMID 40204976›Full record

ArticleCommunications medicine2025

Improvements induced by retinal gene therapy with voretigene neparvovec depend on visual cortical hemispheric dominance mechanisms.

Mariana Ferreira, João Pedro Marques, Miguel Raimundo, Hugo Quental, Miguel Castelo-Branco

Abstract read
In one paragraph

Article in Communications medicine, 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

5 authors.

Mariana Ferreira *Institute of Nuclear Sciences Applied to Health (ICNAS), University of Coimbra, Coimbra, Portugal.ORCID http://orcid.org/0000-0003-2911-7993
João Pedro Marques *Hospitais da Universidade de Coimbra, Unidade Local de Saúde de Coimbra (ULSC), Coimbra, Portugal.ORCID http://orcid.org/0000-0002-1014-0483
Miguel RaimundoInstitute of Nuclear Sciences Applied to Health (ICNAS), University of Coimbra, Coimbra, Portugal.
Hugo QuentalInstitute of Nuclear Sciences Applied to Health (ICNAS), University of Coimbra, Coimbra, Portugal.
Miguel Castelo-BrancoInstitute of Nuclear Sciences Applied to Health (ICNAS), University of Coimbra, Coimbra, Portugal. mcbranco@fmed.uc.pt.ORCID http://orcid.org/0000-0003-4364-6373

Funding

Ministry of Education and Science | Fundação para a Ciência e a Tecnologia (Portuguese Science and Technology Foundation) FCT/UIDB&P/4950/2025
6 · The paper itself

Abstract

backgroundRPE65-associated retinal degeneration (RPE65-RD) causes severe visual deficits. Gene therapy with AAV2-hRPE65v2 is a breakthrough but it is currently unknown which visual pathways benefit from treatment and if cortical mechanisms can amplify retinal improvements.

methodsIn this within-subject design, ten patients with biallelic RPE65-RD underwent sub-retinal injection of AAV2-hRPE65v2. Psychophysical full-field stimulus threshold determination and functional magnetic resonance imaging were performed before and 12 months after treatment. Population receptive fields (pRF) were computed in V1 and visual responses assessed using contrast-reversed checkerboards (3 contrast levels).

resultsHere we show significant improvement in light sensitivity at low-luminance and neural response enhancements under low-luminance conditions specifically in the right hemisphere, which is known to show dominance in attentional and visual pooling of spatial information. Changes in pRF size also reflect known hemispheric spatial asymmetries (left/right biased for local/global analysis, respectively).

conclusionsOur findings show a contribution of known early and high-level cortical dominance mechanisms on improvement, which constrain the effects of therapy and are therefore a target for neurorehabilitation. These findings provide insight into the limits of clinical benefits of gene therapy and suggest that neurorehabilitation approaches may be needed to enhance improvements, similarly to cochlear implants.

Identifiers

PMID40204976
PMCPMC11982196

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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.