Eric E. Swayze

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 paper introduces NMA-modified antisense oligonucleotides that show improved potency compared to MOE-modified SSOs in modulating splicing for SMA and Dravet syndrome.

This paper introduces modified nucleosides in siRNA seed regions to enhance specificity and reduce off-target effects while maintaining on-target activity.

This study characterizes an acute sedation response caused by phosphorothioate ASOs after CNS delivery, showing it is sequence-, dose-, and chemistry-dependent and can be mitigated.

The paper characterizes a transient neurobehavioral response to high-dose oligonucleotide delivery in the CNS and provides a framework to mitigate it through magnesium supplementation.

The paper maps ASO distribution and activity in the CNS of rodents and NHP after central administration, showing widespread effects across cell types.

This study shows that increasing convective forces after intrathecal injection improves rostral distribution of therapeutic ASOs in the CNS of cynomolgus monkeys.

This paper demonstrates that antisense oligonucleotides targeting SNCA reduce alpha-synuclein production, prevent pathology, and show translational potential in non-human primates.