Head to head comparison of target knockdown following OTV vs. anti-TfR:ASO administration in mice

Using single nucleus RNA sequencing, we determined that peripheral administration of OTV:Malat1 results in much more widespread and robust target knockdown throughout the mouse brain compared to anti-TfR:Malat1. TfR.KI mice received four weekly doses of 50mpk OTV:Malat1, anti-TfR:Malat1, Malat1 ASO (at an ASO-equivalent dose), or saline. Tissues were harvested 72 hours after the final dose and snap frozen for molecuar analysis.