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Author

C. Beley

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Open access Aug 2026

Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping

The feasibility of developing a safe and effective lead compound with efficient exon 16 skipping, favorable pharmacokinetic and safety profiles, able to rescue dystrophic transcriptomic and proteomic signatures is demonstrated.

Marine Geoffroy-Guiraud, C. Beley, Stephany Campuzano et al. · 0 citations

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