Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia.

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Title: Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia.
Authors: Lidonnici MR; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. lidonnici.mariarosa@hsr.it., Chianella G; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy., Mende N; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK., Bastos HP; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK., Barcella M; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Merelli I; Institute for Biomedical Technologies, National Research Council, Segrate, Italy., Storto M; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Romeo V; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Tiboni F; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Scaramuzza S; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Rossi C; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Raggi L; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Aprile A; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Crippa S; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy., Iskander D; Department of Immunology & Inflammation, Imperial College London, London, UK., Roberts I; Department of Paediatrics, University of Oxford, Oxford, UK., Karamiditris A; Department of Immunology & Inflammation, Imperial College London, London, UK., Keith J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Lechauve C; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Wilson NK; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK., Göttgens B; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK., Bernardo ME; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy., Ciceri F; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy., Aiuti A; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy., Marktel S; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy., Laurenti E; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK., Ferrari G; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. ferrari.giuliana@hsr.it.; University Vita-Salute San Raffaele, Milan, Italy. ferrari.giuliana@hsr.it.
Source: Nature communications [Nat Commun] 2025 Jul 01; Vol. 16 (1), pp. 5639. Date of Electronic Publication: 2025 Jul 01.
Publication Type: Journal Article
Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia.
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  Data: <searchLink fieldCode="AU" term="%22Lidonnici+MR%22">Lidonnici MR</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. lidonnici.mariarosa@hsr.it.<br /><searchLink fieldCode="AU" term="%22Chianella+G%22">Chianella G</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Mende+N%22">Mende N</searchLink>; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Bastos+HP%22">Bastos HP</searchLink>; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Barcella+M%22">Barcella M</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Merelli+I%22">Merelli I</searchLink>; Institute for Biomedical Technologies, National Research Council, Segrate, Italy.<br /><searchLink fieldCode="AU" term="%22Storto+M%22">Storto M</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Romeo+V%22">Romeo V</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Tiboni+F%22">Tiboni F</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Scaramuzza+S%22">Scaramuzza S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Rossi+C%22">Rossi C</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Raggi+L%22">Raggi L</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Aprile+A%22">Aprile A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Crippa+S%22">Crippa S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Iskander+D%22">Iskander D</searchLink>; Department of Immunology & Inflammation, Imperial College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Roberts+I%22">Roberts I</searchLink>; Department of Paediatrics, University of Oxford, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22Karamiditris+A%22">Karamiditris A</searchLink>; Department of Immunology & Inflammation, Imperial College London, London, UK.<br /><searchLink fieldCode="AU" term="%22Keith+J%22">Keith J</searchLink>; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.<br /><searchLink fieldCode="AU" term="%22Lechauve+C%22">Lechauve C</searchLink>; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.<br /><searchLink fieldCode="AU" term="%22Weiss+MJ%22">Weiss MJ</searchLink>; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.<br /><searchLink fieldCode="AU" term="%22Wilson+NK%22">Wilson NK</searchLink>; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Göttgens+B%22">Göttgens B</searchLink>; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Bernardo+ME%22">Bernardo ME</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ciceri+F%22">Ciceri F</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Aiuti+A%22">Aiuti A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; University Vita-Salute San Raffaele, Milan, Italy.; Pediatric Immunohematology, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Marktel+S%22">Marktel S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy.; Haematology and BMT Unit IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Laurenti+E%22">Laurenti E</searchLink>; Wellcome-MRC Cambridge Stem Cell Institute, Department of Haematology, Jeffrey Cheah Biomedical Centre, University of Cambridge, Cambridge, UK.<br /><searchLink fieldCode="AU" term="%22Ferrari+G%22">Ferrari G</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy. ferrari.giuliana@hsr.it.; University Vita-Salute San Raffaele, Milan, Italy. ferrari.giuliana@hsr.it.
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