Exon skipping approach to recessive dystrophic epidermolysis bullosa
Lay summary
This project uses an approach called exon skipping, which allows mutations (the part of the gene where there are errors) to be skipped, rather than transferring a normal copy of the Collagen VII gene (COL7A1). The aim is to create a shorter but functional Collagen VII protein. Preliminary results have been encouraging in animal models. Now this work will progress to larger proof of concept experiments and toxicology studies prior to a clinical trial in people.
