Gene therapy grants new skin to schoolboy

Berlin, Nov 9 : In a first, a team of doctors successfully treated a seven-year-old boy of a genetic disease, that had destroyed approximately 80 per cent of his skin, using transplants derived from genetically modified stem cells.
Hassan was suffering from "epidermolysis bullosa" -- a congenital skin disease that affects protein-forming genes essential for skin regeneration and currently considered to be incurable.
Even minor stress can result in blisters, wounds, and skin loss with scar formation. Depending on disease severity, internal organs may likewise be affected, leading to critical dysfunctions.
Hassan was admitted to the Children's Hospital at Ruhr-Universitat Bochum in Germany in June 2015 with 60 per cent skin loss.
"He suffered from severe sepsis with high fever, and his body weight had dropped to a mere 17 kgs -- a life-threatening condition," said Tobias Rothoeft, Consultant at the hospital.
The surgeons opted for transplantation of genetically modified epidermal stem cells that was obtained from the patient via skin biopsy.
Eighty per cent of Hassan's skin, including the boy's arms and legs, entire back, flanks, and partially the stomach, neck and face, were transplanted with skin that was high-quality, stress-resistant skin with intact hydrolipid film, as well as early formation of hair, the doctors said.
"Overall, 0.94 square metres of transgenic epidermis were transplanted onto the young patient in order to cover all defects, accounting for 80 per cent of his entire body surface," added Tobias Hirsch, Associate Professor at the varsity's hospital.
Hassan's treatment continued for over a period of eight months, after which he was discharged in February 2016. Hassan has not yet developed any scar contractures in the transplanted areas.
He has also began attending school again and is actively taking part in his family's social life, the doctors said.
Hassan is the first patient worldwide who has been treated with skin transplants from transgenic epidermal stem cells on a large body surface area, they noted.