OpenASO: RNA Rescue--designing splice-modulating antisense oligonucleotides through community science
Victor Tse, Martin Guiterrez, Jill Townley, Jonathan Romano, Jennifer Pearl, Guillermo Chacaltana, Eterna Participants, Rhiju Das, Jeremy R. Sanford, Michael D. Stone
This study uses community science and RNA structure prediction to design splice-modulating ASOs that enhance exon 16 splicing in a hemophilia A model.
RNA · May 27, 2025Peer-reviewedBiochemistry