RT-qPCR Methods to Support Pharmacokinetics and Drug Mechanism of Action to Advance Development of RNAi Therapeutics

Nucleic Acid Ther. 2020 Jun;30(3):133-142. doi: 10.1089/nat.2019.0840. Epub 2020 Mar 23.

Abstract

The goal of this study was to develop a reverse transcription quantitative polymerase chain reaction (RT-qPCR) method for the accurate quantification of chemically modified small interfering RNA (siRNA) including but not restricted to thermally destabilizing modifications such as glycol nucleic acid (GNA). RT-qPCR was found to be superior to mass spectrometry-based siRNA detection in terms of sensitivity and throughput. However, mass spectrometry is still the preferred method when specific metabolite detection is required and is also insensitive to siRNA chemical modifications such as GNA. The RT-qPCR approach can be optimized to take chemical modifications into account and works robustly in different matrices without optimization, unlike mass spectrometry. RT-qPCR and mass spectrometry both have their strengths and weaknesses for the detection of siRNA and must be used appropriately depending on the questions at hand. Considerations such as desired throughput, assay sensitivity, and metabolite identification must be weighed when choosing which methodology to apply.

Keywords: RT-qPCR; pharmacodynamics; pharmacokinetics; small interfering RNA.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Calibration
  • Drug Monitoring / instrumentation
  • Drug Monitoring / methods*
  • Glycols / chemistry
  • Humans
  • Mass Spectrometry
  • Precision Medicine / instrumentation
  • Precision Medicine / methods
  • RNA, Small Interfering / blood
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / pharmacokinetics*
  • RNAi Therapeutics / methods*
  • Reverse Transcriptase Polymerase Chain Reaction / methods*
  • Reverse Transcriptase Polymerase Chain Reaction / standards
  • Sensitivity and Specificity

Substances

  • Glycols
  • RNA, Small Interfering