[Comment] Targeting challenging RDEB skin wounds with gene therapy

Recessive dystrophic epidermolysis bullosa (RDEB) is a systemic disease of poor mucocutaneous basement membrane integrity downstream of bi-allelic pathogenic variants in COL7A1. Cutaneous clinical manifestations include dermal–epidermal separation with minor trauma yielding visible blisters or erosions from birth, poor wound healing, and often early mortality from cutaneous squamous cell carcinoma.1 RDEB care is largely symptom palliation. Although supportive care is crucial, two epidermolysis bullosa-specific therapies have had regulatory approval since 2023: a topical gene therapy using a non-integrating herpes simplex viral vector to deliver full-length COL7A1 to a wound, Vyjuvek,2 and a topical birch bark extract-based immunomodulatory agent to promote keratinocyte migration and wound healing, Filsuvez.

Jun 24, 2025 - 00:00
 0
Recessive dystrophic epidermolysis bullosa (RDEB) is a systemic disease of poor mucocutaneous basement membrane integrity downstream of bi-allelic pathogenic variants in COL7A1. Cutaneous clinical manifestations include dermal–epidermal separation with minor trauma yielding visible blisters or erosions from birth, poor wound healing, and often early mortality from cutaneous squamous cell carcinoma.1 RDEB care is largely symptom palliation. Although supportive care is crucial, two epidermolysis bullosa-specific therapies have had regulatory approval since 2023: a topical gene therapy using a non-integrating herpes simplex viral vector to deliver full-length COL7A1 to a wound, Vyjuvek,2 and a topical birch bark extract-based immunomodulatory agent to promote keratinocyte migration and wound healing, Filsuvez.