NeurosciencebioRxivPreprint
Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain Barrier
Scarlett J. Barker, Mai B. Thayer, Chaeyoung Kim…·Sarah L. DeVos
This paper demonstrates that systemically delivered OTV, a TfR-binding molecule, effectively transports ASO across the BBB, achieving widespread and sustained knockdown of target genes in the CNS of mice and non-human primates.
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