Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system
Hassan H. Fakih, Masahiro Ohara, Ashley Summers, Samantha L Sarli, Karen Kelly, Rosemary Gagnon, Bruktawit Maru, Brianna Bramato, Anastasia Khvorova, Jonathan K. Watts
This study develops D-siRNA, an albumin-binding dendrimer-conjugated siRNA, showing effective and durable gene silencing in the CNS with no detectable toxicity.
Nucleic Acids Research · Sep 15, 2026Peer-reviewedPharmacology & Toxicology