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Article

Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa

7 Feb 2024

Abstract excerpt

Summary Dystrophic epidermolysis bullosa is a rare genetic disease caused by damaging variants in COL7A1 , which encodes type VII collagen. Blistering and scarring of the ocular surface develop, potentially leading to blindness. Beremagene geperpavec (B-VEC) is a replication-deficient herpes simplex virus type 1–based gene therapy engineered to deliver functional human type VII collagen. Here, we report the case...

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PMID: 38324486
DOI: 10.1056/nejmoa2301244

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