Morphological Profiles of RNAi-Induced Gene Knockdown Are Highly Reproducible but Dominated by Seed Effects

PLoS One. 2015 Jul 21;10(7):e0131370. doi: 10.1371/journal.pone.0131370. eCollection 2015.

Abstract

RNA interference and morphological profiling-the measurement of thousands of phenotypes from individual cells by microscopy and image analysis-are a potentially powerful combination. We show that morphological profiles of RNAi-induced knockdown using the Cell Painting assay are in fact highly sensitive and reproducible. However, we find that the magnitude and prevalence of off-target effects via the RNAi seed-based mechanism make morphological profiles of RNAi reagents targeting the same gene look no more similar than reagents targeting different genes. Pairs of RNAi reagents that share the same seed sequence produce image-based profiles that are much more similar to each other than profiles from pairs designed to target the same gene, a phenomenon previously observed in small-scale gene-expression profiling experiments. Various strategies have been used to enrich on-target versus off-target effects in the context of RNAi screening where a narrow set of phenotypes are measured, mostly based on comparing multiple sequences targeting the same gene; however, new approaches will be needed to make RNAi morphological profiling (that is, comparing multi-dimensional phenotypes) viable. We have shared our raw data and computational pipelines to facilitate research.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Cell Line
  • Gene Expression Profiling / methods
  • Gene Knockdown Techniques / methods*
  • Humans
  • RNA Interference*
  • RNA, Small Interfering* / genetics
  • RNA, Small Interfering* / metabolism

Substances

  • RNA, Small Interfering

Grants and funding

The work was supported by the National Science Foundation (NSF CAREER DBI 1148823, to Anne E. Carpenter; NSF RIG DBI 1119830 to Mark-Anthony Bray) and the Slim Initiative for Genomic Medicine, a project funded by the Carlos Slim Foundation in Mexico. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.