Harnessing gene editing to treat rare diseases such as Epidermolysis Bullosa (EB)

Problem

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a rare genetic skin fragility and blistering disorder in which no curative treatment currently exists with palliative care only in use.

Solution

Our proposed solution is to develop a gene editing curative treatment that can be applied topically to wound sites of RDEB patients.

This therapeutic approach will enable the permanent correction of the underlying mutations that give rise to RDEB, in a non-invasive manner.

Team Information

Project Lead

Professor Wenxin Wang, Charles Institute of Dermatology, School of Medicine, University College Dublin

Project Co-applicant

Dr Nan Zhang, School of Mechanical and Materials Engineering, University College Dublin

Societal Impact Champion

Dr Sinead Hickey, Head of Research of DEBRA Ireland

Project Team

Dr. Irene Lara-Sáez, Charles Institute of Dermatology, School of Medicine, University College Dublin

Mr. Jonathan O’Keeffe Ahern, Charles Institute of Dermatology, School of Medicine, University College Dublin