Work

First topical gene therapy treatment for dystrophic epidermolysis bullosa

Year: 2025

Type: other

Abstract: EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the...

Source: Industrias Culturais (Universidade de Coimbra)

DOI: 10.58079/13wg2

Author: Vroby

Language: English

Topic: Skin and Cellular Biology Research

Subfield: Cell Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: green

OpenAlex ID: W6899188286

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