More Steps Towards Treatment
Principal Investigator(s): Professor M. Wood
Institution: University of Oxford
Funding: In collaboration with MDUK
Total grant: £82,173
SMA Trust contribution: £41,086.50 (50%)
Duration: 4 year
Can short molecular “patches” deliver gene therapy without the need for invasive spinal cord injections?
Professor Wood and his team are developing an adapted form of exon skipping technology to treat spinal muscular atrophy (SMA). They are developing short protein fragments, called peptides, to help deliver molecular patches to the brain and spinal cord, eliminating the need for invasive spinal cord injections. This PhD studentship is testing the potential therapeutic approach in mice, with a view to establishing a safe, therapeutic dose – an essential step on the way to clinical trials.