Hope for butterfly skin sufferers: A modified skin graft could alleviate their condition.

Genetically modified skin grafts made from a patient's own cells can repair persistent wounds in people with the extremely painful dermatological condition dystrophic epidermolysis bullosa, also known as "butterfly skin."

This is the main conclusion of a Phase III clinical trial conducted with 11 volunteers and led by Stanford Medicine. In it, researchers demonstrated that patients with this rare disease experienced significantly better healing , less pain, and less itching in wounds treated with these modified grafts, compared to standard care practices.

"With our novel gene therapy technique—described in The Lancet—we have successfully treated the most difficult-to-heal wounds, which used to also be the most painful for these patients ," summarizes Jean Tang, senior author of the study and a scientist at Lucile Packard Children's Hospital at Stanford, in a statement.

When even bathing is painful

Dystrophic epidermolysis bullosa, a type of epidermolysis bullosa, is very rare. Those who suffer from it have a defect in the gene for collagen VII , a protein that normally holds the skin together (without this molecular "staple," the layers of skin separate at the slightest friction, even a light rub).

Wounds are prone to infection, and even bathing is painful. Patients are at high risk of developing skin cancer throughout their lives .

To conduct this trial, researchers individually cultured skin grafts for each patient—all of whom were at least 6 years old.

To do this, they first took a small biopsy of uninjured skin, which they then took to the lab, where they used a retrovirus to introduce a corrected version of the collagen VII gene, COL7AI, into the skin cells.

The genetically modified cells were grown on sheets of skin , each about the size of a credit card. The grafts took about 25 days to prepare, after which a plastic surgeon sutured the genetically modified skin to the wound.

81% of the wounds were healed

According to the results, at 24 weeks after grafting, 81% of treated wounds were at least half healed, compared with 16% of control wounds.

At that same time point, 16% of the grafted wounds had completely healed , compared to none of the controls.

Furthermore, patient reports of pain, itching, and blistering were better in the grafted areas than in the control wounds. The grafts were safe, and the adverse effects experienced were not severe, according to the scientists.