Evidence map›Paper›PMID 41760155›Full record

SynthesisBMJ open2026

Advanced therapeutic approaches for inherited retinal diseases: an umbrella review.

Kingsley Ekemiri, Esther Daniel, Robin Seemongal-Dass, Chioma Ekemiri, Virginia Victor, Maureen Amobi, Onohomo Adebo, Tiwabwork Tekalign

Abstract readSystematic Review
In one paragraph

Synthesis in BMJ open, 2026. 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. Article
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

8 authors.

Kingsley EkemiriDepartment of Optometry, Faculty of Medical Sciences, The University of the West Indies, Tunapuna, Trinidad and Tobago.
Esther DanielSchool of Nursing, Faculty of Medical Sciences, The University of the West Indies, Couva Campus, Trinidad, Trinidad and Tobago.
Robin Seemongal-DassOphthalmology Unit, Department of Clinical an Surgical Sciences, Faculty of Medical Sciences, University of The West Indies, St. Augustine, Trinidad, Trinidad and Tobago.
Chioma EkemiriSchool of Nursing, Faculty of Medical Sciences, The University of the West Indies, Couva Campus, Trinidad, Trinidad and Tobago.
Virginia VictorSchool of Nursing, Faculty of Medical Sciences, The University of the West Indies, Couva Campus, Trinidad, Trinidad and Tobago.
Maureen AmobiDepartment of Optometry, Abia State University, Uturu, Abia State, Nigeria.
Onohomo AdeboDepartment of Optometry, University of Guyana, Georgetown, Guyana.ORCID http://orcid.org/0009-0003-0160-0809
Tiwabwork TekalignSchool of Nursing, collage of medicine and health science, Arba Minch University, Arba Minch, Ethiopia tiwabworkt@gmail.com.ORCID http://orcid.org/0000-0002-4200-4216

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesTo evaluate the efficacy and safety of advanced therapeutic approaches for inherited retinal disease (IRD) using evidence from systematic reviews and meta-analyses.

designUmbrella review. DATA SOURCES: We searched for Epistemonikos, PubMed, Scopus, PsycInfo, Google Scholar, Joanna Briggs Institute Evidence Synthesis, the Cochrane Database of Systematic Reviews and Database of Abstracts of Reviews of Effects from inception to November 2024. ELIGIBILITY CRITERIA: This included English-language systematic review and meta-analysis assessing advanced therapies in patients with IRD (including congenital retinal dystrophies, retinal dystrophies, retinitis pigmentosa (RP), Stargardt disease, X linked RP, achromatopsia, cone-rod dystrophy, choroideraemia and X linked retinoschisis). Reviews that did not meet the methodological quality threshold were excluded. DATA EXTRACTION AND SYNTHESIS: Two reviewers independently screened and extracted the data, with disagreements resolved by consensus. Findings were synthesised narratively due to the substantial overlap of primary studies.

resultSix systematic reviews and meta-analyses published from 2020 onwards were included, comprising between 6 and 21 primary studies per review. The therapies evaluated included gene therapy, cell-based therapy and stem cell-based interventions. Reported effect estimates showed modest to clinically meaningful improvements in best-corrected visual acuity and retinal structural outcomes in selected IRD subtypes, although effect sizes varied widely across interventions and conditions. The GRADE certainty of evidence ranged from moderate to low, reflecting bias, imprecision and heterogeneity risks. Substantial overlap of primary studies was observed (corrected covered area = 28.9%), precluding quantitative pooling across reviews.

conclusionThe findings suggest notable improvements in visual acuity, retinal structure and other critical outcomes, with therapies such as cell therapy, gene therapy and stem cell therapy showing promising results in enhancing treatment efficacy. Although there are examples of successes with supportive evidence, the overall evidence is not sufficiently strong to make general recommendations, as studies still need to be evaluated on a case-by-case basis. Further high-quality, large-scale randomised controlled trials are needed to better confirm their efficacy and safety.

Indexed as

Retinal DiseasesGenetic TherapyHumansMeta-Analysis as TopicSystematic Reviews as TopicInherited retinal diseasesIRDstherapyumbrella review

Identifiers

PMID41760155
PMCPMC12959001

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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.