In Vivo Evaluation of the Three Best-Performing Lipids (113-O14B-3, 113-O16B-3, and 306-O12B-3) for PCSK9-ASO Delivery
The “#” represents the ED50 (mg·kg-1) for different lipids in PCSK9-ASO delivery in vivo. All of the LNPs were composed of cationic lipid, cholesterol, DOPE, and DSPE-PEG2000 (16/4/1/1 w/w) with a PCSK9-ASO-to-lipid ratio of 1/15 (w/w). Animals were treated with single-dose intravenous administration at varying ASO doses (mg·kg-1). Mice were sacrificed after 72 h of postinjection. PCSK9 mRNA levels relative to β-actin mRNA levels were determined in liver samples. Data points are expressed as a percentage of saline control animals and represent group mean ± SD (n = 3).