Combinatorial Synthesis of a Lipidoid Library by Thiolactone Chemistry: In Vitro Screening and In Vivo Validation for siRNA Delivery

Bioconjug Chem. 2020 Mar 18;31(3):852-860. doi: 10.1021/acs.bioconjchem.0c00013. Epub 2020 Feb 28.

Abstract

Transcriptional inhibition by small interfering RNA (siRNA) delivery using synthetic transfection agents eliminates the subsequent risk of introducing mutations in relevant genes, as opposed to viral vectors. However, synthetic vectors with comparable transfection efficiency to that of viral vectors are yet to be developed. Hence, synthesizing new transfection vehicles with low toxicity is important. In this study, a library of lipid-like molecules (lipidoids) was synthesized by thiolactone chemistry. This library facilitated nonviral delivery of siRNA to mammalian cells, inducing sequence-specific knockdown of a target gene. The liposomal nanoparticles complexed with anti-green fluorescent protein (GFP) siRNA were successfully screened for transfection efficiency using a HeLa-GFP cell line. The five best-performing lipidoids identified in the screening were found to exhibit superior GFP-knockdown efficiency compared with commercially available transfection reagents. The efficiency of siRNA delivery by one of these lipidoids with minimal toxicity was further successfully evaluated in vivo using Kdrl:EGFP zebrafish embryos as a model system. Our study would be important as a facile synthetic route of efficient nonviral nucleic acid delivery to live cells and organisms.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Chemistry Techniques, Synthetic
  • Drug Carriers / chemical synthesis*
  • Drug Carriers / chemistry*
  • Drug Carriers / toxicity
  • Gene Silencing
  • HEK293 Cells
  • HeLa Cells
  • Humans
  • Lactones / chemistry*
  • Lipids / chemical synthesis*
  • Lipids / chemistry*
  • Lipids / toxicity
  • Liposomes / chemistry
  • Materials Testing
  • Models, Molecular
  • Molecular Conformation
  • RNA Stability
  • RNA, Small Interfering / chemistry*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism
  • Zebrafish

Substances

  • Drug Carriers
  • Lactones
  • Lipids
  • Liposomes
  • RNA, Small Interfering