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%)

Year: 2014

Duration: 4 year

Status: Ongoing

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.