Dr. Atze Bergsma

• Role: Assistant Professor

Antisense oligonucleotides (ASOs) are a promising therapeutic strategy to treat various neuromuscular diseases (NMDs). For many NMDs, antisense targets have been identified pre-clinically. In our lab we have focused on developing ASOs to treat Pompe disease. We have identified several ASOs that are able to alleviate the aberrant splicing caused by the IVS1 variant, a variant present in >80% of Caucasian patients with Pompe disease. While these findings are promising for the development of a therapy, a major hurdle that still needs to be overcome is the delivery of ASOs to skeletal muscle tissue. The main focus of my work is to develop a delivery strategy that is both safe and effective. We are currently optimizing the use of lipid nanoparticles to deliver antisense oligonucleotides to skeletal muscle tissue.