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Article

Practical considerations relevant to treatment with the gene therapy beremagene geperpavec-svdt for dystrophic epidermolysis bullosa

9 May 2024

Abstract excerpt

Background/purpose Dystrophic epidermolysis bullosa (DEB), a rare genetic skin disease caused by loss-of-function mutations in COL7A1, the gene encoding type VII collagen (COL7), is characterized by skin blistering, scarring, and extracutaneous manifestations that markedly reduce patient quality-of-life. Beremagene geperpavec-svdt (‘B-VEC’) is a gene therapy employing a non-integrating, replication-defective...

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Identifiers

PMID: 38724041
DOI: 10.1080/09546634.2024.2350232

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