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Consensus guidelines for the design and in vitro preclinical efficacy testing N-of-1 exon skipping antisense oligonucleotides
- All authors
- Aartsma-Rus, A.; Garanto, A.; Roon-Mom, W. van; McConnell, E.M.; Suslovitch, V.; Yan, W.X.; Watts, J.K.; Yu, T.W.; N 1 Collaborative
- Date
- 2022-12-13
- Journal
- Nucleic Acid Therapeutics
- Volume
- 33
- Issue
- 1
- Pages
- 17 - 25