Optimising BBB-Penetrant AAV Capsids with Multiplexed, Titrated RNA Payloads for Precise, Systemic Delivery

  • Exploring BBB-crossing AAV capsids for CNS delivery. These capsids enable IV administration for widespread brain transduction while minimising surgery, though peripheral exposure and systemic toxicity remain key challenges
  • Discussing precise control of transgene expression. Neuron-specific promoters and multi-targets RNA payloads maximise CNS gene modulation while reducing total viral load
  • Highlighting multiplexed and titratable RNA strategies. BBB-penetrant AAVs can target multiple genes in a single pathway, and small-molecule regulators allow dynamic modulation for durable, single-dose therapy 

NEW DATA