Gel-Delivered Topical Gene Therapy

Five-year-old Rowan Millner has dystrophic epidermolysis bullosa
From Stanford University: “People with recessive dystrophic epidermolysis bullosa have a genetic mutation that renders them unable to make a protein called collagen VII, which binds the middle and outer layers of the skin together. Without this protein, the layers slide across each other, causing blisters that progress to severely painful open wounds. . . . The gel uses a modified herpes simplex virus to deliver a copy of the collagen VII gene to the surface of the skin, which makes the missing protein and stabilizes the skin’s structure. Because the herpes virus has evolved to evade the human immune system, the gel can be applied repeatedly without triggering an immune response that has stymied previous gene therapies using other viruses to deliver corrective genes to the body.” MORE
Image Credit: Stanford News, Courtesy of the Millner Family