Lipid-siRNA conjugate accesses a perivascular transport mechanism and achieves widespread and durable knockdown in the central nervous system
A. Sorets, K. Schwensen, N. Francini, A. Kjar, A. Abdulrahman, A. Shostak, K. Katdare, K. Schoch, R. Cowell, J. Park, A. Ligocki, W. Ford, L. Ventura-Antunes, E. Hoogenboezem, A. Prusky, , E. Lippmann
This study develops a lipid-siRNA conjugate that uses perivascular transport to achieve long-lasting gene knockdown in the CNS without toxicity.
bioRxiv · Jun 9, 2024 · PreprintBioengineering