Gene therapy could help treat epidermolysis bullosa, a genetic condition in which a person's skin is fragile and easily injured, MIT's Technology Review reports. A clinical trial is underway at Stanford University.
Epidermolysis bullosa can often be traced to due to mutations that prevent patients making type-7 collagen, which would usually hold the epidermis to the underlying dermis, Tech Review says. For Monique Roeder, one of the trial participants, just holding her feet to a card when she was born left blisters, it notes.
For the trial, Stanford's Jean Tang removed two small pieces of skin from Roeder and used a virus to deliver a working type-7 collagen gene to her skin cells. Those cells were then grown into sheets and grafted onto Roeder. Healthy skin has started to form at those graft sites, Tech Review says, and Roeder and trial participants say they can better perform day-to-day tasks without pain. Tang estimates that patients would need new grafts every few years as skin cells are shed.
Tech Review also notes that Abeona Therapeutics has licensed the approach form Stanford.