Doctors save the life of a seven-year-old boy by changing 80% of his skin
srijeda , 01.07.2020.saved his life after receiving a new lab-grown skin.
A seven-year-old boy from Germany who had lost 80% of his epidermis to a genetic disease has
The treatment has for the first time combined stem cell technology to regenerate solid tissue with gene editing technology to correct the defect that causes the disease. Almost two years later, the boy lives a normal life, goes to school, plays soccer, and when he is injured, his skin heals without problems.
"From the experience we have with people treated for burns, we hope that the new epidermis will last a lifetime," said Michele De Luca, co-author of the research, at the Center for Regenerative Medicine at the University of Modena, at a telephone news conference (Italy). "We have treated children, and when they grow up, the stem cells self-regulate and continue to renew the epidermis."
Two years after treatment, the boy goes to school, plays soccer and can lead a normal life.
The boy was admitted in June 2015 to the Bochum University Pediatric Hospital (Germany). He suffered from the crystal skin disease -also known as butterfly skin or epidermolysis bullosa-, which is usually fatal in the first years of life and affects around 500,000 people worldwide.
Since birth, he had had blisters all over his body, especially his arms, legs, back, and sides of the trunk. Her condition quickly worsened in the spring of 2015 following an infection. According to the details of the case presented today in the scientific journal Nature, when I arrived at the hospital I had completely lost the epidermis in 60% of his body.
"We had a hard time keeping him alive in the early days," Tobias Rothoeft, a pediatric intensive care specialist at Bochum Hospital and first author of the research, recalled at the news conference. Doctors tried different treatments but none worked. They contacted other medical teams in Germany, Switzerland and the United States who were unable to provide any solution. They tried to transplant the boy's skin cell pro from her father but her immune system rejected it. "The parents asked us to do everything possible," recalls Rothoeft. But in August, after two months of failure, "we were sure he was going to die."
He arrived at the hospital in critical condition and, after two months of trying to treat him without success, "we were sure that he was going to die," recalls a doctor.
One last option remained. Something that had never been done before. They removed four square centimeters of the child's little healthy skin and in September they sent Michele De Luca to the Center for Regenerative Medicine in Modena. De Luca and his team repaired the genetic defect in the skin cells by introducing them to the correct version of the LAMB3 gene.
Subsequently, they made the cells multiply to obtain layers that could be implanted on the child's body. "It is a technique very similar to the one we have been using in the past to regenerate skin damaged by burns," explains De Luca.
When the layers were ready for the implant in October, the boy had already lost 80% of his epidermis. In a photo published by doctors in Nature with parental permission, her entire body appears red, raw.
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