Functional dystrophin restored by exon skipping
At the end of 2007, Prosensa and the University of Leiden (Netherlands) medical centre team published encouraging results of a first exon skipping trial of antisense oligonucleotides using intramuscular administration. Tolerance of the treatment was good and above all a truncated but functional form of the protein responsible for DMD (dystrophine) was produced in the four patients treated.
Another trial led by Prosensa showed that the same treatment administered subcutaneously allowed antisense oligonucleotides to be distributed throughout the body and dystrophin production was restored in the fifteen patients.