Scientists from Ruhr University for the first time in the history of medicine was able to raise a portion of healthy skin from stem cells of a child suffering from epidermolysis bullosa by editing the faulty gene, and then transplanted to him this “artificial skin”, reports RIA “Novosti” with reference to the journal Nature.
Epidermolysis bullosa – a rare genetic disease in which the skin is very sensitive, vulnerable, unable to withstand even minor trauma, because of what it called the “butterfly syndrome”. On the skin formed blisters and bubbles that often become infected and can lead to malignant tumors in the skin.
This disease is caused by point mutations in the genes encoding the epidermal anchoring proteins (LAMA3, LAMB, LAMC3), is responsible for the connection of the epidermis with the dermis, resulting in between them is broken the mechanical link. But, as found by Tobias Hirsch and his colleagues from Ruhr University, DNA of skin cells can be edited before dividing and eliminate typographical errors in the defective genes that determine the development of diseases of the skin.
German genetics edited by defective genes responsible for disruption of the normal structure of the skin and the development of epidermolysis bullosa. They took stem cells from a healthy area of skin was removed from the DNA of the mutant version of the gene LAMВ3 and with the help of a special retrovirus replaced it with the correct copy.
After the defective genes have been edited from stem cells in a special pattern, mimicking the structure of human skin were grown in the surrounding healthy skin. The researchers were able to grow about 1 sq. m. “artificial skin”, which was transplanted to the patient with epidermolysis bullosa, from whose stem cells it was grown.
Seven year old boy suffering from a severe form of epidermolysis bullosa, by that time already lost around 60% of the skin due to bacterial infections. The boy had two operations, which he replaced about 80% of the skin. After a year of “artificial skin” has fully engrafted. In February of last year, the boy left the clinic and now lives a normal life, just like his peers.
– Now the child is in good health and not suffering from any problems, – commented on the results of this study Cedric Blanpain, a geneticist from the free University of Brussels – however, we need to conduct lengthy observations of his condition, as long as we do not know how such a procedure increases the chances of skin cancer. Likewise, it is unclear how long these cells live, and can they give place to the old stem cells with a mutant version of the gene.