Molecular BiologybioRxivPreprint
Rescue of blood coagulation Factor VIII exon-16 mis-splicing by antisense oligonucleotides
Victor Tse, Guillermo Chacaltana, Martin E Gutierrez…·Jeremy R. Sanford
The paper identifies a three-way junction structure in intron 15 that affects F8 pre-mRNA splicing and shows that antisense oligonucleotides targeting this structure can rescue splicing defects in Hemophilia A.