Megan E. Afetian

This paper develops a VHH-Fc fusion protein to deliver siRNAs and ASOs across the BBB in rodents and NHPs, showing that binding affinity, DAR, and conjugation site affect delivery efficiency.

This study shows that antisense oligonucleotides targeting VCP reduce disease pathology and improve muscle function in cell and mouse models of multisystem proteinopathy.

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.