AtuFECT01

AtuFECT01 is a cationic lipid component. AtuFECT01 is used to construct AtuPLEX/Atu027 Lipid nanoparticles. AtuFECT01 improves cellular uptake and endosomal escape of siRNA in AtuFECT01-based lipoplexes. Formulation of AtuFECT01 with Tie2 siRNA into lipoplexes reduces Tie2 expression in mouse lung^[1][2][3][4]. In Vitro:AtuFECT01-based siRNA-lipoplexes (1-50 nM; 48 h) inhibit PKN3 protein expression in HeLa cells^[2]. In Vivo:AtuFECT01-based siRNA-lipoplex (1.88 mg/kg siRNA and 14.5 mg/kg lipid; i.v. via tail vein; once daily; four consecutive days) reduces endothelial CD31 and Tie2 mRNA/protein in lung, heart, and liver and lowers serum soluble Tie2 in healthy nude mice^[2].
DACC/AtuFECT01 lipoplexes (0.75-3 mg siRNA/kg; intravenous bolus; single or once daily; 3 consecutive days) mediate dose-dependent, lung endothelial-specific knockdown of Tie-2, CD31, VEGFR2, VE-Cadherin, and BMPR-2 in C57Bl/6j healthy mice^[3].