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Gene therapy gives boy his life back

A European medical team has successfully used gene therapy to transform the life of a young boy with serious skin damage.

Scientists from Germany’s Ruhr-Universität Bochum’s burn unit and the Center for Regenerative Medicine at the University of Modena, Italy, carried out the skin transplant treatment using modified stem cells, taken from the patient. Two years on and Hassan, who suffers from epidermolysis bullosa – which had destroyed 80% of his epidermis, is now back in school.

“Transplanting 80% of the skin and providing intensive medical care to the patient over a period of eight months was extremely challenging,” explained Dr Tobias Rothoeft, Ruhr-Universität Bochum. “The close collaboration between the departments in Bochum and the University of Modena’s expertise have been the key to success. This makes us very proud.”

The scientists have published the findings of their work in Nature.

Dr Tobias Hirsch, another key member of the medical team, added: “This approach has enormous potential for research into and development of new therapies for the treatment of epidermolysis bullosa as well as other diseases and trauma causing large skin defects.”

This is a major step in the fight against a congenital skin disease currently considered to be incurable.

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