{"database":"biostudies-literature","file_versions":[],"scores":{"citationCount":0,"reanalysisCount":0,"viewCount":45,"searchCount":0},"additional":{"submitter":["Breveglieri G"],"funding":["FP7 Health","Wellcome Trust"],"pagination":["7426"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7582302"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["21(19)"],"pubmed_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."],"journal":["International journal of molecular sciences"],"pubmed_title":["Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening."],"pmcid":["PMC7582302"],"funding_grant_id":["Innovator Award 208872/Z/17/Z","306201-FP7-HEALTH-2012-INNOVATION-1"],"pubmed_authors":["Breveglieri G","Gambari R","Trapella C","Pacifico S","Borgatti M","D'Aversa E","Zuccato C","Cosenza LC","Guerrini R","Sultan S","Preti D"],"view_count":["45"],"additional_accession":[]},"is_claimable":false,"name":"Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening.","description":"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.","dates":{"release":"2020-01-01T00:00:00Z","publication":"2020 Oct","modification":"2024-10-19T10:15:09.7Z","creation":"2020-10-31T09:57:13Z"},"accession":"S-EPMC7582302","cross_references":{"pubmed":["33050052"],"doi":["10.3390/ijms21197426"]}}