Rare Skin Disorders

Intrexon through collaboration is developing optimal genetic programs to deliver therapies for patients with a rare and devastating skin disorders that often have no treatment or cure.  One such disease is recessive dystrophic epidermolysis bullosa (RDEB).  This autosomal recessive disorder is characterized by the loss of collagen type VII, an important protein component of the anchoring fibers that connect the dermis to the epidermis.  Our proposed treatment for this disease will provide autologous, gene-modified fibroblasts that will produce collagen VII in vivo.