DRUG-seq Provides Unbiased Biological Activity Readouts for Neuroscience Drug Discovery

ACS Chem Biol. 2022 Jun 17;17(6):1401-1414. doi: 10.1021/acschembio.1c00920. Epub 2022 May 4.

Abstract

Unbiased transcriptomic RNA-seq data has provided deep insights into biological processes. However, its impact in drug discovery has been narrow given high costs and low throughput. Proof-of-concept studies with Digital RNA with pertUrbation of Genes (DRUG)-seq demonstrated the potential to address this gap. We extended the DRUG-seq platform by subjecting it to rigorous testing and by adding an open-source analysis pipeline. The results demonstrate high reproducibility and ability to resolve the mechanism(s) of action for a diverse set of compounds. Furthermore, we demonstrate how this data can be incorporated into a drug discovery project aiming to develop therapeutics for schizophrenia using human stem cell-derived neurons. We identified both an on-target activation signature, induced by a set of chemically distinct positive allosteric modulators of the N-methyl-d-aspartate (NMDA) receptor, and independent off-target effects. Overall, the protocol and open-source analysis pipeline are a step toward industrializing RNA-seq for high-complexity transcriptomics studies performed at a saturating scale.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Discovery* / methods
  • Humans
  • RNA
  • Reproducibility of Results
  • Sequence Analysis, RNA / methods
  • Transcriptome*

Substances

  • RNA