In a small study of patients with the rare inherited disease epidermolysis bullosa, researchers have used a DNA-carrying gel to help mend their skin.
They’re known as butterfly children, people born with skin so fragile that a simple touch can create wounds that may never fully heal. Now, in a small study of patients with the rare inherited disease epidermolysis bullosa , researchers have used a DNA-carrying gel to help mend their skin.
Experimental treatments include skin grafts or stem cells engineered to make the protein missing in a specific form of EB. In one dramatic 2017 case, lab-grown sheets of stem cells saved the life of a. But these treatments require hospital stays and anesthesia, and would likely be unaffordable in many countries.
Marinkovich and colleagues first showed treatment with the gel prompted collagen VII production in skin from RDEB patients and mice with the same mutation. In 2018, they launched a clinical trial funded by the company Krystal Biotech Inc. Nine patients, three of them children, had wounds sprinkled with drops of the gel, which was then spread by a bandage. The patients were treated every 1 to 3 days, for 25 days..
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