A Novel High-Content Immunofluorescence Assay as a Tool to Identify at the Single Cell Level γ-Globin Inducing Compounds

PLoS One. 2015 Oct 28;10(10):e0141083. doi: 10.1371/journal.pone.0141083. eCollection 2015.

Abstract

The identification of drugs capable of reactivating γ-globin to ameliorate β-thalassemia and Sickle Cell anemia is still a challenge, as available γ-globin inducers still have limited clinical indications. High-throughput screenings (HTS) aimed to identify new potentially therapeutic drugs require suitable first-step-screening methods combining the possibility to detect variation in the γ/β globin ratio with the robustness of a cell line. We took advantage of a K562 cell line variant expressing β-globin (β-K562) to set up a new multiplexed high-content immunofluorescence assay for the quantification of γ- and β-globin content at single-cell level. The assay was validated by using the known globin inducers hemin, hydroxyurea and butyric acid and further tested in a pilot screening that confirmed HDACs as targets for γ-globin induction (as proved by siRNA-mediated HDAC3 knockdown and by treatment with HDACs inhibitors entinostat and dacinostat) and identified Heme-oxygenases as novel candidate targets for γ-globin induction. Indeed, Heme-oxygenase2 siRNA knockdown as well as its inhibition by Tin protoporphyrin-IX (TinPPIX) greatly increased γ-globin expression. This result is particularly interesting as several metalloporphyrins have already been developed for clinical uses and could be tested (alone or in combination with other drugs) to improve pharmacological γ-globin reactivation for the treatment of β-hemoglobinopathies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Sickle Cell / metabolism
  • Butyric Acid / metabolism
  • Fetal Hemoglobin / metabolism
  • Humans
  • Hydroxyurea / metabolism
  • K562 Cells
  • beta-Globins / analysis*
  • beta-Globins / metabolism
  • beta-Thalassemia / metabolism
  • gamma-Globins / analysis*
  • gamma-Globins / metabolism

Substances

  • beta-Globins
  • gamma-Globins
  • Butyric Acid
  • Fetal Hemoglobin
  • Hydroxyurea

Grants and funding

This work was supported by the People Programme (Marie Curie Actions) of the European Union's Seventh Framework Programme FP7/2007-2013/ under REA grant agreement n°289611 (HEM_ID Project) and by Fondazione Cariplo for grant n° 2012.0517 to AR. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript. FG and IF are employees in Nerviano Medical Sciences S.r.l., which is a private sector partner in the HEM_ID Project (Marie Curie Actions of the European Union's Seventh Framework Programme FP7/2007-2013/ under REA grant agreement n°28961). The present research is the result of a collaborative project and MD is the Marie Curie fellow appointed to Nerviano Medical Sciences S.r.l. within this network. PC was a NMS employee at the time the research was performed (present address: FlowMetric Europe srl, Parco Tecnologico Padano, Via A. Einstein 26900, Lodi, Italy).