Treatment of Epidermolysis Bullosa and Future Directions: A Review.
Blistering
Gene therapy
Repurposed drugs
Skin adhesion
Wound healing
Journal
Dermatology and therapy
ISSN: 2193-8210
Titre abrégé: Dermatol Ther (Heidelb)
Pays: Switzerland
ID NLM: 101590450
Informations de publication
Date de publication:
02 Aug 2024
02 Aug 2024
Historique:
received:
20
05
2024
accepted:
01
07
2024
medline:
2
8
2024
pubmed:
2
8
2024
entrez:
1
8
2024
Statut:
aheadofprint
Résumé
Epidermolysis bullosa (EB) comprises rare genetic disorders characterized by skin and mucosal membrane blistering induced by mechanical trauma. Molecularly, pathogenic variants affect genes encoding proteins crucial for epidermal-dermal adhesion and stability. Management of severe EB is multidisciplinary, focusing on wound healing support, ensuring that patients thrive, and complication treatment. Despite extensive research over 30 years, novel therapeutic approaches face challenges. Gene therapy and protein therapy struggle with efficacy, while regenerative cell-based therapies show limited effects. Drug repurposing to target various pathogenic mechanisms has gained attention, as has in vivo gene therapy with drugs for dystrophic and junctional EB that were recently approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA). However, their high cost limits global accessibility. This review examines therapeutic advancements made over the past 5 years, exploiting a systematic literature review and clinical trial data.
Identifiants
pubmed: 39090514
doi: 10.1007/s13555-024-01227-8
pii: 10.1007/s13555-024-01227-8
doi:
Types de publication
Journal Article
Review
Langues
eng
Informations de copyright
© 2024. The Author(s).
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