Gene therapy breakthrough at University College Dublin

Irish scientists from UCD have announced a breakthrough in the search for a cure for EB. A new solution that uses gene therapy to repair bad cells has been produced. It has the potential to provide a permanent cure for patients with Recessive Dystrophic EB. The concept has been pioneered by Prof. Wenxin Wang, who has been carrying out research into Epidermolysis Bullosa for more than 10 years funded by DEBRA Ireland.

The solution is called “the FedEx of gene editing”, as it removes the need for painful surgery and deliver a biological solution using a simple carrier system.

Dr. Johnathan O’Keeffee Ahern, one of the leading gene therapy researchers on Wang’s team, talked about the breakthrough:

“The best part of this therapy is that it would not involve painful skin grafting procedures or risks associated with viral treatments in delivering funtioning genes to the skin. Rather like using a postcode, we can send in genetic scissors to cut out the non-functioning part of the gene and then the repaired cells know they must fill that space and permanently repair the gene. We would not even need to take the patient cells for skin grafting. The gel would be applied, absorbed and go to work. We have seen some promising results in the lab. With further research, we could expand this to other EB types and even other genetic conditions. We are in the process of forming own spin-off company to continue to develop this technology and take it to the next step, clinical trial.”

EB Researchers from UCD Charles Institute of Dermatology