My research interests focus on the molecular mechanisms underlying inheritable neuromuscular disorders, with a strong emphasis on myotonic dystrophy type 1. I aim to identify novel therapeutic targets and develop interventions to mitigate the heterogeneous symptoms in the patient population. Trained as a molecular cell biologist, I use cells and transgenic mice as model systems. We were among the first to introduce therapeutic antisense oligonucleotides in DM1 and the first to excise the triplet repeat from the DM1 locus in cells using CRISPR/Cas9 technology.
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