Author

B. Ardin

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Open access Sep 2026

Mapping intronic mutational hotspots by in silico mutagenesis enables single antisense oligonucleotide correction of multiple variants

This gene-agnostic framework establishes a systematic strategy for identifying intronic mutational hotspots and matching patients to scalable, mutation-agnostic ASO-based precision therapies.

H. Saei, B. Ardin, N. Kaiser et al. · medRxiv · 0 citations