Delivery of oligonucleotides to bone marrow to modulate ferrochelatase splicing in a mouse model of Erythropoietic Protoporphyria
François Halloy, Pavithra S. Iyer, Paulina Ćwiek, Alice Ghidini, Jasmin Barman‐Aksözen, Nicole Wildner-Verhey van Wijk, Alexandre P. A. Theocharides, Elisabeth Irene Minder, Xiaoye Schneider‐Yin, Daniel Schümperli, Jonathan Hall
This study evaluates splice-switching oligonucleotides (SSOs) in a mouse model of Erythropoietic Protoporphyria, showing that an SSO with a cholesteryl group increases correctly spliced FECH transcript by 80% in bone marrow.
Nucleic Acids Research · Apr 9, 2020Peer-reviewedMolecular Biology