Gene therapy just saved a young boy’s life—by giving him new skin

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Researchers genetically tweaked a seven-year-old boy’s skin cells to fix a disease-causing mutation. Once grown in the lab, the researchers placed them on his body—where they took hold and grew further.
If you regularly read science news, you might think that scientists are obsessed with stem cells. Many of them are, and for good reason: They have an incredible ability to develop into almost any type of cell found in the body. Now that technologies exist to more reliably tweak the human genome, the idea of growing healthy tissues and organs as-needed is sounding less and less like science fiction. In a paper out today in the journal Nature, researchers replaced almost all of a seven-year-old boy’s outer skin layer to treat his life threatening skin condition, a genetic disease called epidermolysis bullosa.
The researchers grew the replacement skin from the boy’s healthy epidermal cells, tweaking their genetic makeup in the lab to ensure the disease-causing mutation wasn’t present. They then attached the new skin to the affected areas of the child’s body. The same group of scientists previously performed a similar procedure on two children with the same condition, but this is the first time they did so on someone with such a severe case. Instead of treating small patches, they replaced 80 percent of his skin surface.
Epidermolysis bullosa prevents the outermost layer of the skin, called the epidermis, from anchoring to the underlying skin layer (the dermis) correctly. As a result, the epidermis falls away when inflicted with even minor trauma, easily causing blisters, ulcers, and in many cases (including the boy in the study) life-threatening wounds on much of the skin surface. Before undergoing the experimental treatment, the young patient described in the study had lost much of his skin to the disease and was admitted to the hospital.
Fortunately, the researchers saw the disease as a prime candidate for their stem cell tweaking technique. They first took a small piece of healthy tissue from an unaffected patch of the boy’s skin. Then, they isolated epidermal cells from that tissue, and using a retroviral vector—essentially a method that viruses use to insert information into a cell—they placed instructions for healthy, non-mutated skin cells into the boy’s own. These cells grew and flourished in the lab, creating the makings of healthy skin. During three surgeries conducted over the course of several months, doctors placed this lab-grown tissue on top of the patient’s dermis so it could continue to grow there.

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