| Literature DB >> 31566258 |
Byungji Kim1, Ji-Ho Park2, Michael J Sailor1,3.
Abstract
With the recent FDA approval of the first siRNA-derived therapeutic, RNA interference (RNAi)-mediated gene therapy is undergoing a transition from research to the clinical space. The primary obstacle to realization of RNAi therapy has been the delivery of oligonucleotide payloads. Therefore, the main aims is to identify and describe key design features needed for nanoscale vehicles to achieve effective delivery of siRNA-mediated gene silencing agents in vivo. The problem is broken into three elements: 1) protection of siRNA from degradation and clearance; 2) selective homing to target cell types; and 3) cytoplasmic release of the siRNA payload by escaping or bypassing endocytic uptake. The in vitro and in vivo gene silencing efficiency values that have been reported in publications over the past decade are quantitatively summarized by material type (lipid, polymer, metal, mesoporous silica, and porous silicon), and the overall trends in research publication and in clinical translation are discussed to reflect on the direction of the RNAi therapeutics field.Entities:
Keywords: RNA interference; drug delivery; gene therapy; nanoparticles; siRNA
Mesh:
Substances:
Year: 2019 PMID: 31566258 PMCID: PMC6891135 DOI: 10.1002/adma.201903637
Source DB: PubMed Journal: Adv Mater ISSN: 0935-9648 Impact factor: 30.849