Syrian Boy Gets Life-Saving Cure from Genetically Grown Skin Experiment

Genetically Modified Skin Grown in a Lab Could Save Syrian Boy

Using human stem cells, researchers from the Free University of Brussels have introduced a new technique to genetically grow human replacement skin that could save the life of a Syrian boy who suffers from a rare genetic disease that is destroying his own skin.  Scientists grew replacement skin for the boy, who, at the time of the surgery, had already lost 80 percent of his skin.  The seven-year old boy is part of an ongoing trial of the new technique.

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From The Guardian

Scientists have grown a replacement, genetically modified skin to cover almost the entire body of a seven-year-old Syrian boy who was suffering from a devastating genetic disorder.

The treatment marks a rare and striking success for the field of regenerative medicine, which has been struggling to transform futuristic-sounding science into therapies that make a difference to patients. In the latest trial, the life of the young boy – whose illness had come close to killing him – was transformed.

Before undergoing surgery, the boy had lost 80% of his skin, leaving him covered in untreatable, infected wounds. He was given morphine to cope with the pain and his doctors were preparing to start palliative treatment after all conventional therapies had failed.

Prof Cédric Blanpain, a stem cell scientist at the Free University of Brussels, described the work as one of the most impressive examples to date of the use of stem cells in humans. “There are very few diseases that have benefitted so far,” he said. “This is a beautiful example of something that was unthinkable before the study. To replace and gene-correct the whole skin of a patient is just amazing.”

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

The boy, who arrived in Germany in 2013 after his family fled Syria as refugees, was suffering from a genetic disease called junctional epidermolysis bullosa, which causes the skin to become fragile and blister. By the time he came to be treated, he had lost the surface layer of skin, called the epidermis, from almost his entire body, with only the skin on his head and a patch on his left leg remaining intact.

His doctors, based at University Children’s Hospital, Ruhr University Bochum, had attempted to graft skin from his father, but the transplant had been rejected. As a last resort, the team sought the help of Italian scientists who had pioneered a technique to regenerate healthy skin in the laboratory – but had never attempted to use it for such an ambitious case.

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