We work at the interface of biotechnology and biomedicine. Our major focus is on RNA biology and RNA therapeutics as applied primarily to rare neuromuscular diseases.
We are interested in the development of RNA therapeutic technologies and in particular technologies for targeted intracellular delivery of RNA therapies (antisense oligonucleotides, gene silencing RNAi therapies and gene editing therapies) to cells and tissues of the neuromuscular system (muscle, heart and brain). These intracellular delivery technologies include development of novel cell-targeting peptides, antibody conjugates and the engineering of extracellular vesicles (EVs) for drug delivery and targeted cellular uptake. Much of our recent work has focussed on Duchenne muscular dystrophy (DMD), spinal muscular atrophy (SMA) and myotonic dystrophy type 1 (DM1) in addition to which we have emerging areas of interest in Huntington’s disease and motor neuron disease (ALS).