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Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.

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Title: Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Authors: Castiello MC; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy., Brandas C; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy., Ferrari S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Porcellini S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Sacchetti N; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Canarutto D; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy., Draghici E; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Merelli I; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy., Barcella M; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy., Pelosi G; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Vavassori V; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Varesi A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Jacob A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Scala S; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Basso Ricci L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Paulis M; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy., Strina D; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy., Di Verniere M; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy., Sergi Sergi L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Serafini M; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy.; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza (MI) 20900, Italy., Holland SM; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Bergerson JRE; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., De Ravin SS; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Malech HL; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Pala F; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Bosticardo M; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Brombin C; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy., Cugnata F; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy., Calzoni E; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy., Crooks GM; Department of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles (UCLA), Los Angeles, CA 90095, USA., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA., Genovese P; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA 02115, USA., Naldini L; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy., Villa A; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.
Source: Science translational medicine [Sci Transl Med] 2024 Feb 07; Vol. 16 (733), pp. eadh8162. Date of Electronic Publication: 2024 Feb 07.
Publication Type: Journal Article; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Extramural
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
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  Data: <searchLink fieldCode="AU" term="%22Castiello+MC%22">Castiello MC</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.<br /><searchLink fieldCode="AU" term="%22Brandas+C%22">Brandas C</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy.<br /><searchLink fieldCode="AU" term="%22Ferrari+S%22">Ferrari S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Porcellini+S%22">Porcellini S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Sacchetti+N%22">Sacchetti N</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Canarutto+D%22">Canarutto D</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.; Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Draghici+E%22">Draghici E</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Merelli+I%22">Merelli I</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy.<br /><searchLink fieldCode="AU" term="%22Barcella+M%22">Barcella M</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; National Research Council (CNR), Institute for Biomedical Technologies, Segrate (MI) 20054, Italy.<br /><searchLink fieldCode="AU" term="%22Pelosi+G%22">Pelosi G</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Vavassori+V%22">Vavassori V</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Varesi+A%22">Varesi A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Jacob+A%22">Jacob A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Scala+S%22">Scala S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Basso+Ricci+L%22">Basso Ricci L</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Paulis+M%22">Paulis M</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy.<br /><searchLink fieldCode="AU" term="%22Strina+D%22">Strina D</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.; Humanitas Clinical and Research Center IRCCS, Rozzano (MI) 20089, Italy.<br /><searchLink fieldCode="AU" term="%22Di+Verniere+M%22">Di Verniere M</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.<br /><searchLink fieldCode="AU" term="%22Sergi+Sergi+L%22">Sergi Sergi L</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Serafini+M%22">Serafini M</searchLink>; Translational and Molecular Medicine (DIMET), University of Milano-Bicocca, Monza 20900, Italy.; Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza (MI) 20900, Italy.<br /><searchLink fieldCode="AU" term="%22Holland+SM%22">Holland SM</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Bergerson+JRE%22">Bergerson JRE</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22De+Ravin+SS%22">De Ravin SS</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Malech+HL%22">Malech HL</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Pala+F%22">Pala F</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Bosticardo+M%22">Bosticardo M</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Brombin+C%22">Brombin C</searchLink>; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Cugnata+F%22">Cugnata F</searchLink>; University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Calzoni+E%22">Calzoni E</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Crooks+GM%22">Crooks GM</searchLink>; Department of Pathology and Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles (UCLA), Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Notarangelo+LD%22">Notarangelo LD</searchLink>; Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD 20892, USA.<br /><searchLink fieldCode="AU" term="%22Genovese+P%22">Genovese P</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA 02115, USA.<br /><searchLink fieldCode="AU" term="%22Naldini+L%22">Naldini L</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Vita-Salute San Raffaele University, Milan 20132, Italy.<br /><searchLink fieldCode="AU" term="%22Villa+A%22">Villa A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCSS San Raffaele Scientific Institute, Milan 20132, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica (IRGB), Consiglio Nazionale delle Ricerche (CNR), Rozzano (MI) 20089, Italy.
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