296 Nanoneedles for gene editing in Epidermolysis Bullosa

Abstract

Recessive Dystrophic Epidermolysis Bullosa is an autosomal recessive disorder caused by loss of function mutations in COL7A1 causing mucocutaneous blistering and scarring, with considerable morbidity and mortality. A few early phase gene therapy trials have been reported as potential therapeutic approaches. The targeted nature of base editing is well suited to restore COL7A1 functionality in RDEB, if supported by an effective delivery strategy. Nanoneedles are a “bed of nail” substrate that interface tightly with cells for gene transfer in primary, hard to transfect cells and exposed tissue layers.