Publication - Abstract
Sep 29, 2020
Nature Communications
November 16, 2014
The discovery of RNA interference (RNAi) in mammalian cells has created a new class of therapeutics based on the reversible silencing of specific disease-causing genes. This therapeutic potential depends on the ability to deliver inducers of RNAi, such as short-interfering RNA (siRNA) and micro-RNA (miRNA), to cells of target tissues. This chapter reviews various challenges and delivery strategies for siRNA, with particular focus on the development of lipid nanoparticles (LNP) delivery technologies. Currently, LNP delivery systems are the most advanced technology for systemic delivery of siRNA, with numerous formulations under various stages of clinical trials. We also discuss methods to improve gene silencing potency of LNP-siRNA, as well as application of LNP technologies beyond siRNA to the encapsulation of other nucleic acids such as mRNA and clustered regularly interspaced short palindromic repeats (CRISPR).
Publication - Abstract
Sep 29, 2020
Nature Communications
Publication - Abstract
Oct 14, 2015
Chemical Reviews
Described first in the 1960s by Bangham1 and understood as a potential drug delivery system in the early 1970s,2−4 the liposome has since become integral to research and clinical applications in the field of nanomedicine.