My DPhil project is largely focused on applying CRISPR/Cas9 genome engineering for the molecular correction of Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). In addition to this, I am working on developing combination therapies for such neuromuscular disorders whereby the muscle architecture is stabilised and the genetic cause of the disease can be targeted simultaneously.
Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1.
Marsh S. et al, (2020), Mol Ther, 28, 2527 - 2539
Mutation-independent proteomic signatures of pathological progression in murine models of Duchenne muscular dystrophy.
van Westering TLE. et al, (2020), Mol Cell Proteomics
Extracellular microRNAs exhibit sequence-dependent stability and cellular release kinetics.
Coenen-Stass AML. et al, (2019), RNA Biol, 16, 696 - 706