Science & Technology



9-year-old boy with rare disease now has engineered skin covering 80 percent of his body

Melissa Healy, Los Angeles Times on

Published in Science & Technology News

For a soccer-playing, holiday-loving boy on the brink of death from a rare inherited skin disease, doctors have accomplished a feat of genetic engineering, surgical stamina and raw nerve.

Infections arising from blisters and sores had destroyed 60 percent of his skin. Just 7 years old when he arrived at a burn unit in Bochum, Germany, the child was in excruciating pain despite round-the-clock morphine. He suffers from junctional epidermolysis bullosa, or JEB, a disorder that is generally fatal in the first few years of life. Though the boy had outlived most with the disease, it clearly appeared that he was about to die.

A team of German and Italian physicians had a bold idea to spare him that grim fate.

Over a five-month period in the fall and winter of 2015, they harvested some of the boy's few remaining healthy skin cells.

Using a virus to invade the cells, they introduced a corrected version of the mutated gene that had caused the catastrophic failure of his epidermis, the body's largest organ.

They cultured the corrected cells and, on sheets backed with plastic and a naturally occurring adhesive, used them to build many square meters of healthy new epidermis.

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Then, ever so delicately, they clothed his small body in a new set of skin.

More than two years after being admitted to a burn unit of the Ruhr University Children's Hospital, the 9-year-old boy has downy hairs pushing through his smooth skin. He runs and plays with other children, gets bruises and blisters that heal normally, and needs no drugs or daily ointments to protect or preserve his transplanted outer layer of cells.

A report on his cutting-edge treatment was published Wednesday by the journal Nature.

For some of the roughly half a million children worldwide afflicted with JEB, the report brings real hope. A pioneering therapy could extend survival beyond the first few years of life, and might even restore normal skin function in some.


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