Self-degradable “gemini-like” ionizable lipid-mediated delivery of siRNA for subcellular-specific gene therapy of hepatic diseases
Qiu Wang, Bin Wan, Yao Feng, Zimeng Yang, Dan Li, Fan Liu, Ya Gao, Chang Li, Yanhua Liu, Yongbing Sun, Zhonggui He, Cong Luo, Jin Sun, Qikun Jiang
Abstract
-acyltransferase 2 (DGAT2) in the hepatocytes. Our findings provide new insights into developing novel highly effective and low-toxic "gemini-like" ionizable lipids for constructing LNPs, potentially achieving more effective treatment for hepatic diseases.
Topics & Concepts
Genetic enhancementChemistryGene deliverySmall interfering RNAGeneCell biologyBiophysicsBiochemistryBiologyTransfectionRNA Interference and Gene DeliveryVirus-based gene therapy researchPancreatic function and diabetes