As reported by NPR, a child in Europe who was born with a rare genetic disorder was on the verge of death until gene therapy reversed the disease.
In this disease, children are born with a flawed gene that prevents the outer layer of the skin, the epidermis, from binding to the inner layer. This can cause excruciating blisters to form all over these children’s bodies.
The child needed more than 60% of is skin to be replaced. Doctors had decided it was a lost clause when his doctors learned of researchers in Italy who were experimenting with a new treatment for this disease. Michele De Luca and colleagues at the University of Modena and Reggio Emilia were genetically engineering skin cells to repair the genetic flaw.
De Luca used a virus to insert a healthy gene into cells taken from the boy’s skin. Some of those cells, stem cells, multiply indefinitely. So De Luca was able to grow entire sheets of engineered epidermis, which were shipped to the hospital in Germany.
The treatment worked and the young boy is now doing well.
Around 25,000 people in the U.S. suffer from this same excruciating and debilitating skin condition (epidermolysis bullosa) and an estimated 500,000 worldwide.
This is such great news for those sufferers and their families. And the area of gene therapy is an area that holds so much promise for further development to treat a variety of serious and chronic conditions.