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.
Latest research
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The International GBS Outcome Study (IGOS)
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Erasmus MC
Duration: May 4, 2012 – December 2, 2024 -
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The International CIDP Outcome Study (ICOS): An Update
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Amsterdam UMC (location AMC)
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Erasmus MC
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UMC Utrecht
Duration: November 12, 2015 – December 31, 2026 -
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Training in the 3D Muscle-on-a-Chip Model
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Erasmus MC
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Leiden UMC
Duration: July 1, 2023 – July 1, 2027 -