Gene Therapy Gel Heals Wounds Caused by "Butterfly-Disease"

There’s a new gel containing DNA that could potentially be used as a treatment for “butterfly disease” which is a condition where blisters form on the skin whenever pressure is applied to it. A small trial involving six adults and three children with the disease. They carried a subtype of the disease recessive dystrophic epidermolysis bullosa (RDEB). This means that their cells are unable to build the protein collagen VII. This protein binds several layers of skin together which prevents the layer from painfully rubbing against each other. This creates the blisters and wounds that could take months or even years to heal. 

There are a plethora of other ongoing treatments for this disease which involves skin grafts and engineered stem cells. Compared to these treatments, gel therapy can be applied much easier and based on the results of the trial, it is more effective. The gel therapy involves applying a gel based ointment onto the patients’ skin. The gel contains a virus that has been modified so that it cannot replicate in the cell. The trial took place over 25 days. A placebo was placed on the wound of the patients as well as the gel therapy ointment. There was no change in the wounds  that the placebo was applied on and some even reopened. The wounds will have been healed after applying the gel therapy ointment within about three months.

This form of treatment is extremely useful because while it doesn’t specifically treat the disease, it treats the wounds that form as a result of them. Unfortunately, these wounds and blisters last for a long period of time. This new ointment isn’t a cure to the disease, but it directly treats the  wounds that form because of the disease. These results are still pretty new and they haven’t yet been published in full. It will be interesting to see if it gets approved by the FDA within the next year.