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2022
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antisense oligonucleotides
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toxicity
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Goyenvalle, A.
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Aartsma-Rus, A.
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Networking to optimize Dmd exon 53 skipping in the brain of mdx52 mouse model
Considerations in the preclinical assessment of the safety of antisense oligonucleotides
Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice
Delivery is key: lessons learnt from developing splice-switching antisense therapies
Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy