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Successful gene therapy for “butterfly children” in Salzburg

A biopsy was carried out on the patient, in which epidermal stem cells were isolated in a special laboratory of the Center for Regenerative Medicine in Modena, Italy. The scientists implanted a healthy LAMB3 gene in the stem cell at the laboratory in Modena. The healthy gene ensures that the cells can once again produce functional LAMB3 protein. Skin transplants were produced from the corrected stem cells. 

The skin of “butterfly children” is as sensitive as the wings of a butterfly. The therapeutic success raises hopes for patients, especially people with serious forms of this previously incurable skin disease. In the course of the last year the transplants of the 50-year-old healed very well. A chronic sore with a size of 10x15 centimeters located on the patient’s right lower leg could be completely closed. According to doctors, the patient suffered for a decade from the sore which did not heal.  

At the present time, this type of therapy is only applicable for just a few individuals suffering from EB. However, scientists are working feverishly on correction molecules for other gene mutations.

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