235: experimental therapy heals US toddler

Today's linked story from USA Today is disturbing, but incredible too, and ultimately full of hope. It's about a young boy called Nate from New Jersey, USA, who has inherited a rare genetic disorder, called epidermolysis bullosa. So has his older brother. Both of them are unable to make a protein called collagen VII, which helps skin tissue bind to muscle. The consequences are pretty unpleasant - blistering, scarring, internal tissue damage and more. However, Nate recently benefited from some experimental therapy, involving a bone marrow transplant from a healthy brother. It's early days, but this risky procedure appears to have had a tremendous impact. Nate is now putting on weight and even making collagen VII - in other words, the transplant appears to have helped his body override the genetic deficit he inherited. There is some hope that Nate's older brother may benefit too, though his bone marrow match was not perfect, making the procedure even riskier. Nate's family and experts seem to agree, however, that this is a new therapy that holds tremendous promise.