Your Cells and Our Science: Gene Therapy for Dystrophic Epidermolysis Bullosa


Dystrophic epidermolysis bullosa (DEB) is a progressive, devastatingly painful and debilitating genetic disorder, and one of the most chronic and severe types of epidermolysis bullosa (EB). Two major types of DEB are described based on the autosomal inheritance pattern: recessive DEB (RDEB) and dominant DEB (DDEB).  Both are caused by mutations within the type VII collagen gene (COL7A1).

Research into the underlying genetic mutations that cause DEB has advanced considerably in recent years leading to new opportunities to create personalized biologics with disease-modifying potential.