Rescue of blood coagulation Factor VIII exon-16 mis-splicing by antisense oligonucleotides
Victor Tse, Guillermo Chacaltana, Martin E Gutierrez, Nicholas M. Forino, Arcelia Gonzalez Jimenez, Hanzhang Tao, H. Phong, Catherine Oh, Priyanka Chary, Isabel Quesada, Antonia Hamrick, Sophie Hyesuk Lee, Michael D. Stone, 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.
bioRxiv · Mar 31, 2023 · PreprintMolecular Biology