Evidence map›Paper›PMID 40455038›Full record

ArticleTranslational vision science & technology2025

Characterizing Inner Retinal Changes in End-Stage Inherited Retinal Diseases That Might be Suitable for Optogenetic Therapies.

Benjamin W J Ng, Tien-En Tan, Vasil Kostin, Robert E MacLaren, Jasmina Cehajic-Kapetanovic

Abstract read
In one paragraph

Article in Translational vision science & technology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

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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

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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

2 citing papers in PubMed.

  1. Review
  2. 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.

Benjamin W J NgOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.
Tien-En TanOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.
Vasil KostinOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.
Robert E MacLarenOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.
Jasmina Cehajic-KapetanovicOxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Purpose: The purpose of this study was to characterize retinal structure in patients with late-stage inherited retinal diseases (IRD) for their suitability for optogenetic gene therapy. Methods: This was a retrospective study using clinical data and spectral-domain optical coherence tomography (SD-OCT) images of patients with late-stage IRD (visual acuity ≤ 1.0), between December 2012 and 2023 from Oxford Eye Hospital, United Kingdom. Depending on the clinical phenotype and history, the patients were divided into three groups: rod-cone dystrophy (group 1), cone-rod/cone dystrophy (group 2), and macular dystrophy (group 3). SD-OCT structural parameters including total subfoveal thickness and, if possible, individual inner layers thickness were analyzed. Results: 36 patients with late-stage IRD (11, 13, and 12 in groups 1, 2, and 3) and 54 eyes (18 per group) with mean age of 55.9 ± 9.8 years and mean visual acuity of 1.72 ± 0.66 were analyzed. Mean subfoveal thickness was reduced to 167.8 ± 54.3, 153.2 ± 65.3, and 138.1 ± 41.7 µm in groups 1, 2, and 3, respectively, with no significant difference among each group (P = 0.33). Twenty-five of 54 eyes had well-defined inner retinal layers with mean subfoveal thickness of nerve fiber, ganglion cell, inner plexiform, and inner nuclear layers were 12.6 ± 3.9, 17.3 ± 9.9, 18.6 ± 6.7, and 29.4 ± 11.3 µm, respectively. Conclusions: In our cohort, 46.3% of degenerate retinae had preservation of the inner retina, including nerve fiber, ganglion cell, and inner plexiform layers, and/or thickening of the inner nuclear layer and may benefit from targeted cell-specific optogenetic gene therapy. Patients with indiscernible or disrupted inner layers may be amenable to a non-cell-specific approach, to target all surviving neurons. Translational Relevance: SD-OCT structural characterization of different groups of late-stage IRD offers insight into vector selection and patient eligibility for optogenetic treatments.

Indexed as

Cone-Rod DystrophiesGenetic TherapyMacular DegenerationOptogeneticsRetinaRetinal DiseasesAdultAgedFemaleHumansMaleMiddle AgedRetrospective StudiesTomography, Optical CoherenceVisual Acuity

Identifiers

PMID40455038
PMCPMC12136128

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