(ORDO NEWS) — A new gel containing DNA could help people deal with an extremely rare but debilitating condition called epidermolysis bullosa, or “butterfly syndrome.”
“Butterfly Syndrome” – a disease in which wounds, blisters and erosion appear on the skin even from the most minor injuries
Butterfly Syndrome, or epidermolysis bullosa, is an inherited condition that causes the skin to become so fragile that it can break at the slightest touch, like a butterfly’s wings.
The disease manifests itself from an early age. Affected children have mutations in the COL7A1 gene, which encodes collagen VII. Collagen VII is a vital structural protein that supports the skin.
One of the ways to treat the “butterfly syndrome” is a stem cell transplant. However, this invasive method requires skin grafting, which is expensive and requires anesthesia and hospitalization.
In a new study published in the journal Nature Medicine , scientists have turned to gene therapy to add a working copy of COL7A1 back into the skin cells of people with the disease.
The team first conducted trials in mice and cultured cells before moving on to clinical trials.
The study involved 31 children and adults suffering from “butterfly syndrome”. The treatment was carried out with the help of a gel that was applied to the skin in certain areas.
After 12 weeks, 70% of the wounds treated with the gel had healed. In comparison, in the placebo control group, healing was observed in only 20% of participants.
The study also did not reveal any serious side effects.
Now the scientists are going to apply to the US Food and Drug Administration to get approval for the gel treatment.
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