Enhancement of splicing efficiency between trans‐spliced DMD AAV vectors for the production of full‐length fully functional dystrophin protein.

This is follow-on work from Dr Keith Foster who demonstrated that the triple-transplicing method of gene delivery does work in vitro and Professor Dickson’s will investigate this work further.

The aim of this proposal is therefore to use alternative strategies to improve the splicing efficiency between trans-splicing AAV vectors for the delivery of the DMD gene. The strategies, being equally applicable to both dual and triple trans splicing vectors for mini and full- ength DMD gene delivery, and also to the planned next generation of trans‐splicing vectors.