Gene therapy breakthrough at University College Dublin
Irish scientists from the 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 bilogical solution using a simple carrier system.
Dr. Johnathan O’Keeffee Ahern, one oft he leading gene therapy researchers at Wang’s team 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 a genetic scissors to cut out the non-functioning part of the gene and then the repaired cells know to fill that space and permanently reapir the gene. We would not need to even 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-out company to continue to develop this technology and take it to the next step, a clinical trial.“