Application of gene therapies to inherited skin disorders
In collaboration Dr Wei Li Di, Dr Jemima Mellerio and Professor John Harper's dermatology research group, lentiviral gene therapy is being developed for the inherited skin disease, Netherton Syndrome. Patients with this condition will be the first to be treated with engineered epidermal sheets. The trial is now open and recruiting.
Working with Professsor John McGrath at Guys Hospital, we are also developing similar reagents for the debilitating blistering skin disorder Epidermolysis Bullosa (EB) and have produced a lentiviral platform for the expression of COL7A1 in EB fibroblasts. A clinical trial of engineered fibroblasts should start in 2015.