Werke:
- A gene gun-mediated nonviral RNA trans-splicing strategy for Col7a1 repair
- Splicing modulation via antisense oligonucleotides in recessive dystrophic epidermolysis bullosa
- COL7A1 editing via RNA trans-splicing in RDEB-derived skin equivalents
- A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa