Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease
- Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease
- Optimising CNS delivery of therapeutic payloads using TXP1, a novel second‑generation, brain‑selective shuttle engineered for enhanced transcytosis across the BBB
- Identifying a potent MAPT‑targeting oligonucleotide for tau silencing and optimising its site‑specific conjugation to TXP1 to enable efficient CNS delivery and robust knockdown
- Demonstrated in vivo efficacy, with potent MAPT knockdown observed in a PoC study in transgenic mice following intravenous administration of TXP1‑MAPT