Omics-assisted therapy development
- Validate antisense oligonucleotide (AO)-mediated improvement of muscle quality in vivo
- Validate therapeutic applicability of AO-mediated exon skipping for LGMD2B in vivo
- Show proof of concept for exon skipping of caspase cleavage domains as a therapy for PolyQ diseases
- Assess the omics effects of compounds that induce hyperglycosylation for CMD