Evidence map›Paper›PMID 42866341›Full record

ArticleAnnals of medicine and surgery (2012)2026

Emerging gene therapies in the treatment of recessive dystrophic epidermolysis bullosa: a systematic review.

Abolfazl Nikkhoy, Amirhossein Larijani, Erfan Rajabi, Hojat Eftekhari

Abstract read
In one paragraph

Article in Annals of medicine and surgery (2012), 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

4 authors.

Abolfazl NikkhoyStudent Research Committee, Guilan University of Medical Sciences, Rasht, Iran.
Amirhossein LarijaniStudent Research Committee, Guilan University of Medical Sciences, Rasht, Iran.ORCID https://orcid.org/0000-0002-6240-2756
Erfan RajabiStudent Research Committee, Iran University of Medical Sciences, Tehran, Iran.
Hojat EftekhariDepartment of Dermatology, Skin Research Center, Razi Hospital, School of Medicine, Guilan University of Medical Sciences, Rasht, Iran.ORCID https://orcid.org/0000-0002-4325-5551

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Recessive dystrophic epidermolysis bullosa (RDEB) is a life-limiting collagen type VII alpha-1 (COL7A1)-related disorder characterized by skin fragility, chronic wounds, and early mortality. Gene therapies aim to restore collagen type VII (C7) expression and improve wound healing, but clinical evidence remains limited. Objective: To synthesize current evidence on the safety and effectiveness of gene therapy approaches for RDEB. Methods: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, we searched major databases through 7 March 2025 for interventional studies evaluating gene therapies in RDEB. Two reviewers independently performed study selection, data extraction, and risk-of-bias assessment. Given the substantial heterogeneity, findings were synthesized narratively without meta-analysis. Results: Five clinical trials met the inclusion criteria and evaluated Conclusion: Gene therapy for RDEB demonstrates a favorable early safety profile and promising efficacy. However, the small number of heterogeneous early-phase trials and limited follow-up preclude definitive conclusions. Larger, rigorously designed studies with extended follow-up are required to confirm long-term safety and clinical effectiveness.

Indexed as

epidermolysis bullosa dystrophicagenetic therapysystematic review

Identifiers

PMID42866341
PMCPMC13657577

What Socratic holds

Textmetadata
Read underepoch 390

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 Socratic graph.