September 18, 2013
Technological advances in both siRNA (small interfering RNA) and whole genome sequencing have demonstrated great potential in translating genetic information into siRNA-based drugs to halt the synthesis of most disease-causing proteins. Despite its powerful promises as a drug, siRNA requires a sophisticated delivery vehicle because of its rapid degradation in the circulation, inefficient accumulation in target tissues and inability to cross cell membranes to access the cytoplasm where it functions. Lipid nanoparticle (LNP) containing ionizable amino lipids is the leading delivery technology for siRNA, with five products in clinical trials and more in the pipeline. Here, we focus on the technological advances behind these potent systems for siRNA-mediated gene silencing.
Publication - Abstract
Nov 26, 2020
Journal of Controlled Release
mRNA was formulated in lipoplexes with ionizable, cationic or zwitterionic complexing lipids.
Cell populations in human skin were analyzed for RNA uptake and expression over 72 h.
Adipocytes exhibit...
Publication - Abstract
Aug 11, 2020
Journal of Controlled Release
siRNA and mRNA structures were separated from the ChriST mRNA by RNase H-mediated cleavage of RNA/DNA ...