Enteral siRNA delivery technique for therapeutic gene silencing in the liver via the lymphatic route.

ABSTRACT: An efficient targeting delivery technology is needed for functional oligonucleotides to exert their potential effect on the target gene without an adverse effect in vivo. Development of enteral delivery systems for nucleic acids is a major challenge because of their large molecular size and instability. Here, we describe a new enteral delivery technique that enables small interfering RNA (siRNA) selectively delivered to the liver to silence its target Apolipoprotein B gene expression. A nuclease-resistant synthetic siRNA was conjugated with α-tochopherol and administered as lipid nanoparticle to the large intestine of the mice in a postprandial state. The selective transport into the liver, effective gene silence, and consequently significant reduction in serum low density lipoprotein-chol

SUBMITTER: Murakami M 

PROVIDER: S-EPMC4655470 | biostudies-literature | 2015 Nov

REPOSITORIES: biostudies-literature