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- W3091936764 abstract "The screening of chemical libraries based on cellular biosensors is a useful approach to identify new hits for novel therapeutic targets involved in rare genetic pathologies, such as β-thalassemia and sickle cell disease. In particular, pharmacologically mediated stimulation of human γ-globin gene expression, and increase of fetal hemoglobin (HbF) production, have been suggested as potential therapeutic strategies for these hemoglobinopathies. In this article, we screened a small chemical library, constituted of 150 compounds, using the cellular biosensor K562.GR, carrying enhanced green fluorescence protein (EGFP) and red fluorescence protein (RFP) genes under the control of the human γ-globin and β-globin gene promoters, respectively. Then the identified compounds were analyzed as HbF inducers on primary cell cultures, obtained from β-thalassemia patients, confirming their activity as HbF inducers, and suggesting these molecules as lead compounds for further chemical and biological investigations." @default.
- W3091936764 created "2020-10-15" @default.
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- W3091936764 date "2020-10-08" @default.
- W3091936764 modified "2023-09-26" @default.
- W3091936764 title "Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening" @default.
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- W3091936764 doi "https://doi.org/10.3390/ijms21197426" @default.
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