ArticleAnnals of medicine and surgery (2012)2026
Emerging gene therapies in the treatment of recessive dystrophic epidermolysis bullosa: a systematic review.
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.
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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.
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4 authors.
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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.
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