Gene therapy could be a treatment for genetic inherited diseases such as recessive dystrophic epidermolysis bullosa (RDEB), a condition where the skin lacks a structurally important molecule called type VII collagen and the skin blisters and wounds easily.
The main goal of Dr Wenxin Wang's team is to use a non-viral gene delivery system to place a functional version of the full-length gene COL7A1 in the skin directly, so that skin cells called keratinocytes can make type VII collagen where it is needed.
The team have carried out preliminary work in keratinocytes that lack the ability to make type VII collagen. When the gene was introduced into a standard plasmid (pcDNA3.1COL7A1), the cells made full-length chains of type VII collagen.
They are also working with another, preferred approach called minicircle DNA (MC) to see if using these DNA molecules to deliver the COL7A1 gene - and tweaking them with molecules called S/MARs - could result in skin cells making type VII collagen for longer periods.
When Wang's group have determined arrangements of the DNA delivery molecule and the gene itself, they will analyse where the therapeutic COL7A1 gene goes when it is applied to skin using their delivery system.