Boy gets brand new skin with gene therapy

He had a rare and incurable skin disease caused by genetic mutations

Doctors treating a critically ill boy with a devastating skin disease used experimental gene therapy to create an entirely new skin for most of his body in a desperate attempt to save his life.

Two years later, the doctors report the boy is doing so well that he doesn’t need any medication, is back in school and even playing football.

“We were forced to do something dramatic because this kid was dying,” said Dr. Michele De Luca of the University of Modena in Italy, who got a call for help from the German doctors treating the boy.

The boy, then 7, was hospitalised in June 2015 with blisters on his limbs, back and elsewhere. He quickly lost about 60 per cent of the outer layer of his skin and was put into an induced coma to spare him further suffering. Doctors at Children’s Hospital at Ruhr University in Bochum, Germany, tried skin grafts from his father and donor skin, but all failed. The boy’s parents asked about experimental treatments, and De Luca and his colleagues were contacted.

The boy had a rare, incurable skin disease called junctional epidermolysis bullosa, caused by genetic mutations.

To fix that, the doctors took a small piece of the boy’s skin from an area that was OK. In the lab, they added a normal version of his bad gene to his skin cells. They grew sheets of the boy’s skin, in much the same way skin grafts are grown for burn victims. In total, they grew close to a square metre of skin. –AP