Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics
University of British Columbia · BC Children's Hospital · +2 more institutions
Abstract
Delivering nucleic acid-based therapeutics to cells is an attractive approach to target the genetic cause of various diseases. In contrast to conventional small molecule drugs that target gene products (i.e., proteins), genetic drugs induce therapeutic effects by modulating gene expression. Gene silencing, the process whereby protein production is prevented by neutralizing its mRNA template, is a potent strategy to induce therapeutic effects in a highly precise manner. Importantly, gene silencing has broad potential as theoretically any disease-causing gene can be targeted. It was demonstrated two decades ago that introducing synthetic small interfering RNAs (siRNAs) into the cytoplasm results in specific…
Citation impact
- FWCI
- 15.12
- Percentile
- 100%
- References
- 56
Authors
5- JAJayesh A. Kulkarni
University of British Columbia, BC Children's Hospital
- DWDominik Witzigmann
University of British Columbia
- SCSam Chen
University of British Columbia
- PRPieter R. CullisCorresponding
University of British Columbia
- RVRoy van der Meel
University of British Columbia, University Medical Center Utrecht, Eindhoven University of Technology
Topics & keywords
- Translation (biology)
- Nanoparticle
- Nanotechnology
- Chemistry
- Materials science
- Biochemistry
- Messenger RNA