Antiviral Efficacy of a Short PNA Targeting microRNA-122 Using Galactosylated Cationic Liposome as a Carrier for the Delivery of the PNA-DNA Hybrid to Hepatocytes

摘要: Peptide nucleic acids (PNAs) that bind to complementary acid sequences with extraordinarily high affinity and sequence specificity can be used as antisense oligonucleotides against microRNAs, namely antagomir PNAs. However, methods for efficient cellular delivery must developed effective use of PNAs therapeutic agents. Here, we demonstrate delivered hepatic cells by DNA oligonucleotide cationic liposomes containing galactosylated ceramide a novel lipid, DMKE (O,O'-dimyristyl-N-lysyl glutamate), through glycoprotein-mediated endocytosis. An PNA was designed target miR-122, which is required translation the hepatitis C virus (HCV) genome in hepatocytes, hybridized complexation liposome. The PNA-DNA hybrid molecules were efficiently internalized into complexing liposome vitro. Galactosylation significantly enhanced both lipoplex cell binding cells. After 4-h incubation lipoplexes, HCV suppressed more than 70% sequestration miR-122 cytoplasm. readily released from low pH environment endosome. present study indicates transfection using an non-viral carrier targeted hepatocytes.