Noninvasive imaging of lipid nanoparticle-mediated systemic delivery of small-interfering RNA to the liver.

ABSTRACT: Mouse models with liver-specific expression of firefly luciferase were developed that enable a noninvasive and longitudinal assessment of small-interfering RNA (siRNA)-mediated gene silencing in hepatocytes of live animals via bioluminescence imaging. Using these models, a set of lipid nanoparticles (LNPs) with different compositions of cationic lipids, polyethylene glycol (PEG), and cholesterol, were tested for their abilities in delivering a luciferase siRNA to the liver via systemic administration. A dose-dependent luciferase knockdown by LNP/siRNA assemblies was measured by in vivo bioluminescence imaging, which correlated well with the results from parallel ex vivo analyses of luciferase mRNA and protein levels in the liver. RNA interference (RNAi)-mediated target silencing was furthe

SUBMITTER: Tao W 

PROVIDER: S-EPMC2956923 | biostudies-literature | 2010 Sep

REPOSITORIES: biostudies-literature