Discovery of a new selective ERAP1 inhibitor for Hedgehog-dependent cancer treatment.

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Title: Discovery of a new selective ERAP1 inhibitor for Hedgehog-dependent cancer treatment.
Authors: Bufalieri F; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Cucinotta A; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Cammarone S; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy., Agnoli F; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Basili I; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy; Institut Curie, PSL Research University, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France; Université Paris-Saclay, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France., Ferri G; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Quaglio D; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy., Caimano M; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Capriotti AL; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy., Montone CM; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy., Lospinoso Severini L; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Tempora P; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy., D'Amico S; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy., Juretich F; Department of Scienze del Farmaco, University of Piemonte Orientale, 28100 Novara, Italy., Semrau MS; Protein Targets for Drug Discovery Lab, Elettra Sincrotrone Trieste S.C.p.A, 34149 Basovizza, Trieste, Italy., Navacci S; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Conenna M; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Bordone R; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Spallotta F; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy; Department of Biology and Biotechnologies 'Charles Darwin', Sapienza University of Rome, 00185 Rome, Italy., Garavaglia S; Department of Scienze del Farmaco, University of Piemonte Orientale, 28100 Novara, Italy., Storici P; Protein Targets for Drug Discovery Lab, Elettra Sincrotrone Trieste S.C.p.A, 34149 Basovizza, Trieste, Italy., Ghirga F; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy., Laganà A; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy., Locatelli F; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy., Ayrault O; Institut Curie, PSL Research University, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France; Université Paris-Saclay, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France., Infante P; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy., Botta B; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy., Mori M; Department of Biotechnology, Chemistry, and Pharmacy, University of Siena, 53100 Siena, Italy. Electronic address: mattia.mori@unisi.it., Fruci D; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy. Electronic address: doriana.fruci@opbg.net., Di Marcotullio L; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy. Electronic address: lucia.dimarcotullio@uniroma1.it.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2026 Jan 07; Vol. 34 (1), pp. 582-605. Date of Electronic Publication: 2025 Oct 17.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Discovery of a new selective ERAP1 inhibitor for Hedgehog-dependent cancer treatment.
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  Data: <searchLink fieldCode="AU" term="%22Bufalieri+F%22">Bufalieri F</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Cucinotta+A%22">Cucinotta A</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Cammarone+S%22">Cammarone S</searchLink>; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Agnoli+F%22">Agnoli F</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Basili+I%22">Basili I</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy; Institut Curie, PSL Research University, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France; Université Paris-Saclay, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France.<br /><searchLink fieldCode="AU" term="%22Ferri+G%22">Ferri G</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Quaglio+D%22">Quaglio D</searchLink>; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Caimano+M%22">Caimano M</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Capriotti+AL%22">Capriotti AL</searchLink>; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Montone+CM%22">Montone CM</searchLink>; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Lospinoso+Severini+L%22">Lospinoso Severini L</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Tempora+P%22">Tempora P</searchLink>; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22D'Amico+S%22">D'Amico S</searchLink>; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Juretich+F%22">Juretich F</searchLink>; Department of Scienze del Farmaco, University of Piemonte Orientale, 28100 Novara, Italy.<br /><searchLink fieldCode="AU" term="%22Semrau+MS%22">Semrau MS</searchLink>; Protein Targets for Drug Discovery Lab, Elettra Sincrotrone Trieste S.C.p.A, 34149 Basovizza, Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Navacci+S%22">Navacci S</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Conenna+M%22">Conenna M</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Bordone+R%22">Bordone R</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Spallotta+F%22">Spallotta F</searchLink>; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy; Department of Biology and Biotechnologies 'Charles Darwin', Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Garavaglia+S%22">Garavaglia S</searchLink>; Department of Scienze del Farmaco, University of Piemonte Orientale, 28100 Novara, Italy.<br /><searchLink fieldCode="AU" term="%22Storici+P%22">Storici P</searchLink>; Protein Targets for Drug Discovery Lab, Elettra Sincrotrone Trieste S.C.p.A, 34149 Basovizza, Trieste, Italy.<br /><searchLink fieldCode="AU" term="%22Ghirga+F%22">Ghirga F</searchLink>; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Laganà+A%22">Laganà A</searchLink>; Department of Chemistry, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Locatelli+F%22">Locatelli F</searchLink>; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Ayrault+O%22">Ayrault O</searchLink>; Institut Curie, PSL Research University, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France; Université Paris-Saclay, INSERM U1330/CNRS EMR 8001, Children's Oncology Research Unit (CONCERT), Equipe Labellisée Ligue Contre le Cancer, Orsay, France.<br /><searchLink fieldCode="AU" term="%22Infante+P%22">Infante P</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Botta+B%22">Botta B</searchLink>; Department of Chemistry and Technology of Drugs, Sapienza University of Rome, 00185 Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Mori+M%22">Mori M</searchLink>; Department of Biotechnology, Chemistry, and Pharmacy, University of Siena, 53100 Siena, Italy. Electronic address: mattia.mori@unisi.it.<br /><searchLink fieldCode="AU" term="%22Fruci+D%22">Fruci D</searchLink>; Bambino Gesù Children's Hospital, IRCCS, 00146 Rome, Italy. Electronic address: doriana.fruci@opbg.net.<br /><searchLink fieldCode="AU" term="%22Di+Marcotullio+L%22">Di Marcotullio L</searchLink>; Department of Molecular Medicine, Sapienza University of Rome, 00161 Rome, Italy; Istituto Pasteur Italia-Fondazione Cenci Bolognetti, Sapienza University of Rome, 00161 Rome, Italy. Electronic address: lucia.dimarcotullio@uniroma1.it.
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