Exon Skipping with U7 gene transfer
Action Duchenne is funding a project that aims to instruct the muscle cells to produce themselves the antisense oligoribonucleotides, AONs,which are necessary for exon skipping, so that they do not have to be applied repeatedly. This can be achieved by transporting into the muscles the genetic information for the construction of the AONs. This approach will use U7-snRNAs, small nuclear RNAs, which have a structure similar to splicing factors. These U7-snRNAs can be modified so that they are able to cause exon skipping. The U7 gene will be transported by AAV viral vectors constructs that have been shown in the mdx mouse and Golden retriever dog models to produce dystrophin protein in muscle cells.
This project will be conducted at the University of Oxford with Aurelie Goyenvalle and Professor Kay Davies.