Scientists saved a boy in a critical condition by growing him an entirely new genetically modified skin.
The seven-year old boy was affected by an inherited disease known as epidermolysis bullosa. This disease causes skin to blister and tear at the lightest touch and can be fatal in children.
Scientists took a tiny sample of skin from the boy and used genetic modification to remove the gene defect triggering the condition. They then grew it into a film that was large enough to cover his entire body.
Dr Michele de Luca, from the University of Modena, Italy, who led the gene therapy team, said: “The patient was in danger of life. The prognosis was very poor, but he survived. He went back to normal life, including school and sports. His epidermis is stable; robust. It doesn’t blister at all and functionality is quite good.”
After 21 months, it appeared that the boy has full recovered with his skin no longer blistering. Details of the treatment which had previously been used to reconstruct small areas of skin in two patients have been published in the latest issue of scientific journal Nature.
“The different forms of epidermolysis bullosa affect approximately 500,000 people worldwide. The successful outcome of this study paves the way for gene therapy to treat other types of epidermolysis bullosa and provides a blueprint that can be applied to other stem cell-mediated ex-vivo (outside the body) cell and gene therapies.”
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