Molecular BiologyNucleic Acids ResearchPeer-reviewed
Delivery of oligonucleotides to bone marrow to modulate ferrochelatase splicing in a mouse model of Erythropoietic Protoporphyria
François Halloy, Pavithra S. Iyer, Paulina Ćwiek…·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.
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