A TLS11a-decorated ionizable lipid nanoparticle platform and a multilevel-validated CRISPR LDLR-knockout HepG2 model for hepatocyte-preferential mRNA delivery
A modular aptamer-guided LNP platform to enable hepatocyte-selective genome editing through an LDLR-independent route is designed and validated and a validated LDLR-null hepatocyte model is validated for studying LDLR-dependent biology and disease.