title: A qRT-PCR Method for Determining the Biodistribution Profile of a miR-34a Mimic.
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.
authors.affiliation: Mirna Therapeutics, 2150 Woodward Street, Suite 100, Austin, TX, 78744, USA, kkelnar@mirnarx.com.
authors.forename: Kevin
authors.initials: K
authors.lastname: Kelnar
authors.forename: Andreas G
authors.initials: AG
authors.lastname: Bader
mesh_terms: D000818:Animals; D008081:Liposomes / Q000378:metabolism; D035683:MicroRNAs / Q000235:genetics / Q000378:metabolism*; D011323:Primates / Q000235:genetics; D020133:Reverse Transcriptase Polymerase Chain Reaction / Q000379:methods*; D013223:Statistics as Topic; D014018:Tissue Distribution
chemical_list: D008081:Liposomes; D035683:MicroRNAs