Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous system
Alexander G. Sorets, Katrina R Schwensen, Nora Francini, Andrew Kjar, Adam M Abdulrahman, Alena Shostak, Ketaki A. Katdare, Kathleen M. Schoch, Rebecca P. Cowell, Joshua C. Park, Alexander P Ligocki, William T Ford, Lissa Ventura‐Antunes, Ella N. Hoogenboezem, Alex Prusky, , Ethan S. Lippmann
This study develops a lipid-siRNA conjugate that uses perivascular transport to achieve long-lasting gene knockdown in the CNS without toxicity.
Nucleic Acids Research · Jun 20, 2025Peer-reviewedBioengineering