Cas12n-DOTAP lipid nanoparticle

Cas12n-DOTAP lipid nanoparticle is a non-viral gene editing delivery system developed by Castalysis Bioscience. It consists of the Cas12n nuclease and a single guide RNA (sgRNA) encapsulated within a lipid nanoparticle (LNP) that incorporates the cationic lipid DOTAP (1,2-dioleoyl-3-trimethylammonium-propane). The addition of DOTAP (typically at a ratio of 0.5% to 2%) facilitates the formation of stable complexes with the negatively charged gene editing components through electrostatic interactions. This formulation is designed to enhance cellular uptake, endosomal escape, and nuclear localization of the Cas12n/sgRNA complex. Preclinical studies in cellular models, mice, and non-human primates (NHPs) have demonstrated that the incorporation of DOTAP significantly improves gene editing efficiency compared to conventional four-component LNPs by improving intracellular trafficking and reducing degradation of the nuclease complex.

Other names

DOTAP-incorporated Cas12n LNPCas12n/sgRNA-DOTAP LNP