Robert J Lee
The Ohio State University, USA
Title: Lipid nanoparticles for delivery of therapeutic oligonucleotides
Biography
Biography: Robert J Lee
Abstract
Oligonucleotides, including antisense oligos, siRNA, miRNA mimics, anti-miRs, are promising as therapeutic agents because of their ability to regulate expression of specific or networks of genes that are critical in human diseases. However, clinical translation of oligonucleotide therapeutics has had limited success partly due to their limited nuclease stability and obstacles in their in vivo delivery, especially to tissues other than the liver. To address these issues, a combination of chemical modifications and lipid nanoparticle (LNP)-based delivery strategy has been developed. Design of LNPs needs to balance the requirements of stability in circulation and ability to facilitate intracellular delivery. Additional consideration is needed to address hematological biocompatibility and effects on the immune system. Specific examples will be provided on several novel LNP formulations for therapeutic delivery of miR-29b mimics, anti-miR21, and antisense oligos to Akt-1 in murine tumor models.