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 01, 2015
Clinical Cancer Research
Lipid nanoparticle (LNP) formulations facilitate tumor uptake and intracellular processing through an enhanced permeation and retention effect (EPR), and currently multiple products are undergoing clinical evaluation.
Publication - Summary
Jul 13, 2017
Cell
Lately there has been intense effort to develop vaccines for Zika virus, in part due to the recently established link between Zika infection and congenital birth defects such as microcephaly and the rapid spread of Zika in Oceania and the Americas ...