New Scientist explains a gene therapy procedure that saved the life of a young Syrian boy suffering from a severe skin condition. The gene therapy corrected a genetic mutation responsible for the painful and dangerous skin-blistering disease. The condition is known as Epidermolysis bullosa. The condition is extremely rare. It causes sores by the slightest bit of contact with skin. The life expectancy of people with the mutation is rather short lived because of the complications associated with the sores.
What could cause such a agonizing disease? Genetic mutations. The mutation of the gene forces the surface skin layer away from the underlying layer, resulting in the painful skin sores. Michele DeLuca, of the University of Modena, was able to use gene therapy to save a child that was suffering so severely. At the age of seven, the patient was admitted to a hospital due to an infection that resulted in loss of most of his skin. DeLuca, with help from a team of experts, removed a patch of skin from the patient and genetically modified the cells which provided a correction of the mutation. "They then grew the cells into sheets of skin, which were grafted onto the boy’s body, covering around 80 per cent of him" (Nature, DOI: 10.1038/nature24487). Now, at the age of nine, the boy is able to carry on with a normal and healthy life.
I think this is an absolutely amazing find. This poor young boy was living his life in such pain and misery. To have a small portion of skin genetically modified, resulting in a corrected genetic mutation is ground breaking in my eyes. Science is advancing so quickly, more and more cures for diseases are bound to surface eventually. It's so exciting to see what medicine of the future has in store for the world.