Two-year-old Nate Liao has spent his young life swathed in bandages from head to toe.Nate has a rare and deadly genetic disease that prevents his skin from attaching to his body. The slightest friction against his skin, such as the rubbing of the seam from his shirt, gave him blisters the size of water balloons. Swallowing anything but baby food tore his esophagus.
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For the first time, doctors say, they appear to have cured the disease Nate has, called epidermolysis bullosa.
"Every now and then, you really feel like you've done something great," says John Wagner, a hematologist at the University of Minnesota Medical School who oversaw Nate's therapy, a bone marrow transplant from his healthy 3-year-old brother, Julian. Doctors did the transplant to give Nate a vital protein that he was born without. That protein, collagen VII, glues the outermost layer of skin to the underlying layer, Wagner says.
The goal was for stem cells from the transplanted bone marrow to travel to Nate's skin and begin to make collagen VII once they arrive, says Angela Christiano of Columbia University, who identified the gene for Nate's disease and collaborated on his therapy.
USA Today