A critically ill boy affected by a rare and severe disease got a new chance of survival after a gene therapy was used to produce skin to cover a big part of his body. The technique borrowed the common practice of growing skin grafts for transplant, and mixed it with genetic engineering.
The skin disease has many manifestations
The boy suffered from junctional epidermolysis bullosa, a genetic mutation which affects the skin. However, the disease can have many manifestations, as there are many mutations which might cause it. About 25,000 Americans suffer from it, while the condition affects around 500,000 people from all over the world.
So far, the condition seemed incurable, but the new technique proved revolutionary. However, it might not work for everybody. Since the condition is so diverse and expresses itself in so many ways, it’s hard to engineer a cure to work for all of these manifestations.
However, this treatment was created for the boy, and did wonders for his condition. Even if it cannot apply to everyone, this is the first time when an effective therapy is found for the condition. Also, it was a premiere in terms of skin transplant as well. It was the first successful attempt to cover such a big surface of the body with transplanted cells.
Researchers engineered the mutated gene and grew skin grafts containing it
The researchers had to hurry with the administering of the treatment, as the boy’s condition was so severe that he wasn’t expected to survive for long. After they collected a sample of the boy’s skin, they picked up the gene which caused the condition, engineered a non-mutated form, and then put it back in the DNA.
Afterwards, they could grow skin grafts containing this healthy gene, and applied it over the boy’s body. The procedure was quite long and slow, and the boy had to spend eight months into hospital. In the end, the researchers could confirm the effectiveness of the technique. The study has been published in the journal Nature.
Image Source: Pixabay