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Boy With Rare Disease Gets Genetically Altered Skin

10 November 2017, 10:12 am EST By Samriddhi Dastidar Tech Times
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A seven-year-old boy in Germany was given a new genetically modified skin, which covers 80 percent of his body, in a series of lifesaving operations in 2015.

Now, two years later, the doctors have reported that the patient, named Hassan, is doing so well that he has rejoined school and is even participating in soccer games.

Butterfly Skin

The child was diagnosed with a genetic disease known as junctional epidermolysis bullosa, which makes the skin as fragile as the wings of a butterfly. He developed blisters on his back, limbs, and elsewhere, losing about 60 percent of his skin’s outer layers.

Hassan was hospitalized at Children’s Hospital in Bochum’s Ruhr University, where doctors had reportedly put him into an induced coma to spare him from further suffering. The doctors then tried skin grafts from the child’s father and donor skin but that failed to bring about a solution.

After the child’s parents asked about experimental treatments, the doctors treating Hassan contacted Dr. Michele de Luca of the University of Modena in Italy, whose team had conducted a similar experiment previously used gene therapy to produce a small piece of skin.

De Luca, however, warned the family that Hassan’s risky state meant that he might not live through the complicated operations needed to save him.

“It was a tough decision for us, but we wanted to try for (our son),” Hassan’s father said.

De Luca and his team took a piece of Hassan’s skin for observation, subsequently repaired its DNA in the laboratory, and successfully grafted back the modified skin on the boy. To date, no problems have been detected. The doctor has said that the research team will observe Hassan closely for any signs of skin cancer and other probable issues.

“For the first time, outside the [blood-producing] system, it was able to show that transgenic stem cells can permanently regenerate an entire tissue,” De Luca said. “All the clinical, biological and molecular parameters are fine, his epidermis is stable, robust, doesn’t blister at all and its functionality is quite good.”

Success Of The Surgery And Its Implications

The surgery’s success offers hope to people all over the world who suffer from junctional epidermolysis bullosa, as well as those who have similar skin conditions. The operation has reportedly also thrown a positive light on the fact that genetically engineered cells can be integrated with human tissues, even at large scales.

The details of the case were published on Nov. 10 in the journal Nature.

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