A qRT-PCR Method for Determining the Biodistribution Profile of a miR-34a Mimic

Methods Mol Biol. 2015:1317:125-33. doi: 10.1007/978-1-4939-2727-2_8.

Abstract

MRX34 has recently entered the clinic as the first therapeutic product based on a microRNA (miRNA) mimic. In order to measure drug concentrations in vivo, a quantitation method is needed that exhibits high precision, accuracy, and robustness. While most clinical applications for oligonucleotide therapeutics involve methods based on hybridization assays and liquid chromatography-tandem mass spectrometry, quantitative PCR (qPCR) is a less well-described approach. Here, we present an RT, qPCR, and analysis method to determine the tissue biodistribution of endogenous as well as a therapeutic, exogenous miRNA mimic therapeutic. Assay performance is demonstrated on multiple tissues from nonhuman primates dosed with MRX34.

MeSH terms

  • Animals
  • Liposomes / metabolism
  • MicroRNAs / genetics
  • MicroRNAs / metabolism*
  • Primates / genetics
  • Reverse Transcriptase Polymerase Chain Reaction / methods*
  • Statistics as Topic
  • Tissue Distribution

Substances

  • Liposomes
  • MicroRNAs