Oral presentation
pre-mRNA splicing, RNA splicing, RNA structure, Antisense oligos, disease
Public Summary
Discovery of antisense oligonucleotides (ASOs) that rescue RNA splicing of an array of Hemophilia A (HA) linked pathogenic variants by targeting an inhibitory RNA structure. These findings reveal a rationale for developing a RNA-based therapy for HA.
Presenter
Victor Tse
Graduate Student
Jeremy Sanford, University of California Santa Cruz