Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain.

ABSTRACT: Manipulation of gene expression in the brain is fundamental for understanding the function of proteins involved in neuronal processes. In this article, we show a method for using small interfering RNA (siRNA) in lipid nanoparticles (LNPs) to efficiently silence neuronal gene expression in cell culture and in the brain in vivo through intracranial injection. We show that neurons accumulate these LNPs in an apolipoprotein E-dependent fashion, resulting in very efficient uptake in cell culture (100%) with little apparent toxicity. In vivo, intracortical or intracerebroventricular (ICV) siRNA-LNP injections resulted in knockdown of target genes either in discrete regions around the injection site or in more widespread areas following ICV injections with no apparent toxicity or immune reactions

SUBMITTER: Rungta RL 

PROVIDER: S-EPMC3889191 | biostudies-literature | 2013 Dec

REPOSITORIES: biostudies-literature