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

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

AbstractBackground/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 qua...

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Main Authors: Amy S. Paller (Author), Shireen V. Guide (Author), Diego Ayala (Author), Mercedes E. Gonzalez (Author), Anne W. Lucky (Author), Isin Sinem Bagci (Author), M. Peter Marinkovich (Author)
Format: Book
Published: Taylor & Francis Group, 2024-12-01T00:00:00Z.
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