Child Given Life-Saving Modified Skin

Those who suffer from EB lack collagen the part of the skin that keeps the outer layer known as the epidermis and the inner layer called the dermis together

He was given morphine to cope with the pain and his doctors were preparing to start palliative treatment after all conventional therapies had failed.

Grown in the laboratory, the repaired cells produced colonies of regenerative "mother" stem cells. "That's the price we have to pay for advanced therapy for a genetic disease", De Luca says. "This is a attractive example of something that was unthinkable before the study". No one had ever attempted using gene therapy to replace as much skin as the boy needed, however, and De Luca's work had been sidelined by bureaucratic hurdles.

Claire Higgins, a lecturer of bioengineering at Imperial College London, described the trial as "a huge achievement and quite remarkable".

The pioneering surgery was performed by scientists to save the boy from the life-threatening condition junctional epidermolysis bullosa (JEB), which causes skin to blister and tear at the slightest touch. By the time he came to be treated, he had lost the surface layer of skin, called the epidermis, from nearly his entire body, with only the skin on his head and a patch on his left leg remaining intact.

The boy had been transferred to the burns unit at Ruhr-University, Bochum Children's Hospital in June 2015 with most of his epidermis - the outermost layer of skin - missing or horribly damaged.

A period of four months inpatient recovery then followed, and Hassan was discharged in February 2016, the team said: "Transplanting 80% of the skin and providing intensive medical care to the patient over a period of eight months was extremely challenging".

Dr Michele de Luca, from the University of Modena, Italy, who led the gene therapy team, said: "The patient was in danger. the prognosis was very poor, but he survived". This matches up with the experience of burn victims who receive skin grafts, according to de Luca.

Epidermolysis Bullosa is a genetic condition caused by a defect in protein-forming genes that are essential for skin regeneration, and is considered to be incurable. Without this protein the skin blisters easily, causing chronic wounds and ulcers to form.

Details of the treatment, previously only used to reconstruct small areas of skin in two patients, appear in the latest issue of Nature.

The Italian scientists used a virus to insert a healthy version of the rogue Lamb3 gene into cells taken from the skin tissue sample. This also allows scientists to grow grafts in culture, simply by taking a small sample.

In total, they grew close to a square meter of skin (9 square feet.) The lab-grown skin was then transplanted onto the boy in three operations, ultimately covering 80 percent of his body. Within a month, the graft had integrated into the lower layers of skin. Then, they used a benign virus to genetically modify the stem cells within the skin to make laminin-322 and grew almost a full square meter of the skin.

Writing in Nature, the scientists said: "Transgenic epidermal stem cells can regenerate a fully functional epidermis virtually indistinguishable from a normal epidermis". "All the data we have ... are telling us that this is going to be a stable situation". In a younger child who is less critically ill, still-healthy epidermis can be removed much more slowly and replaced in pieces over the course of a year or two, he says.

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 soccer.

The operation's success offers hope to people worldwide who suffer from JEB, and the many others who experience similar skin conditions.

Treating the boy had provided useful scientific information that improved understanding of how skin was regenerated and maintained, said the scientists.

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