A Dying Boy Gets a New, Gene-Corrected Skin

Ed Yong in The Atlantic:

Lead_960 (1)At the age of 7, Hassan had already seen more than his fair share of hardship. A week after he was born in Syria, a blister appeared on his back. The doctors there diagnosed him with a genetic disorder called epidermolysis bullosa, or EB, which leaves one’s skin extremely fragile and prone to tearing. There was no cure, they said. When Hassan’s family fled Bashar al-Assad’s regime and moved to Germany as refugees, the doctors there said the same thing. Meanwhile, the blisters were getting bigger.

In June 2015, Hassan was admitted to the burn unit of a children’s hospital in Bochum, Germany. By that time, around 60 percent of his epidermis—the top layer of his skin—was gone. His back, flanks, and limbs had become a continuous landscape of open wounds, red and raw. Much of it was badly infected. The pain was excruciating. “Why do I have to live this life?” he asked his father.

Five weeks later, Hassan’s doctors had run out of options, and were planning to start end-of-life care. But after his father asked about experimental treatments, they contacted Michele de Luca, a stem-cell biologist at the University of Modena and Reggio Emilia. Over the past decades, de Luca had been working on a way of giving EB patients fresh skin. He would collect stem cells from their body, edit the faulty genes that were causing their condition, use the corrected cells to grow healthy epidermis, and graft these new layers back onto the patients.

More here.