This could be wonderful news.
The disease, which affects roughly 300 people in Ireland, causes severe blistering from even minor internal or external trauma. In its most severe forms, it can be fatal. "The best part of this therapy is that it would not involve painful skin grafting procedures or risks associated with viral treatments in delivering functioning genes to the skin,” said Dr. Jonathan O’Keeffe Ahern, one of the leading gene therapy researchers on the team.
"Rather like using a postcode, we can send in a genetic scissors to cut out the non-functioning part of a gene and then the repaired cells know to fill that space and permanently repair the gene."If the cure does indeed work, it could take the form of something as simple as a gel. Following their latest success, the treatment will now move forward to clinical trial, says O'Keeffe Ahern.