Epidermolysis bullosa, also known as ‘Butterfly Children’ disease, in reference to the highly delicate nature of the skin of children with this disorder, is a condition linked to a mutation in 16 different genes.
One traditional and expensive solution has been stem-cell therapy, the gel developed avoids this, and can be applied regularly to wound dressing. The gel contains a therapeutic gene that is spread directly onto the skin. “The treatment works by replacing the gene that encodes for collagen VII (C7), which is a protein missing in those with recessive dystrophic epidermolysis bullosa (RDEB).”
C7 is responsible for holding the dermis and epidermis together. Those who lack C7, experience painful blisters, scarring, and sometimes even skin cancers.
The gel treatment 'injects' genetically modified fibroblasts directly into the skin, and does so via the cold sore virus, herpes simplex type 1 (HSV-1).
HSV-1 is capable of infecting skin cells and can evade our immune systems. After genetically engineering the virus so it cannot replicate and spread to other parts of the body, researchers used HSV-1 to carry two genetic variants that code for C7 into the skin.
The gel is currently at the end of phase III clinical trials, where the results are being published; however, so far 67% of the patients treated with this gel, experienced complete healing.