Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.

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Title: Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.
Authors: Castiello MC; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Di Verniere M; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Draghici E; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Fontana E; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Clinical and Research Center, IRCCS, Rozzano, Milan, Italy., Penna S; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Sereni L; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Zecchillo A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Minuta D; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Uva P; Clinical Bioinformatics, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Zahn M; ProtaGene CGT GmbH, Heidelberg, Germany., Gil-Farina I; ProtaGene CGT GmbH, Heidelberg, Germany., Annoni A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Iaia S; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Ott de Bruin LM; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, Netherlands.; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Notarangelo LD; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands., Villa A; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy., Capo V; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
Source: Frontiers in immunology [Front Immunol] 2023 Nov 13; Vol. 14, pp. 1268620. Date of Electronic Publication: 2023 Nov 13 (Print Publication: 2023).
Publication Type: Journal Article; Research Support, Non-U.S. Gov't; Research Support, N.I.H., Intramural
Journal Info: Publisher: Frontiers Research Foundation] Country of Publication: Switzerland NLM ID: 101560960 Publication Model: eCollection Cited Medium: Internet ISSN: 1664-3224 (Electronic) Linking ISSN: 16643224 NLM ISO Abbreviation: Front Immunol Subsets: MEDLINE
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  Data: Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.
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  Data: <searchLink fieldCode="AU" term="%22Castiello+MC%22">Castiello MC</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Di+Verniere+M%22">Di Verniere M</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Draghici+E%22">Draghici E</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Fontana+E%22">Fontana E</searchLink>; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.; Humanitas Clinical and Research Center, IRCCS, Rozzano, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Penna+S%22">Penna S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Sereni+L%22">Sereni L</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Zecchillo+A%22">Zecchillo A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Minuta+D%22">Minuta D</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Uva+P%22">Uva P</searchLink>; Clinical Bioinformatics, IRCCS Istituto Giannina Gaslini, Genoa, Italy.<br /><searchLink fieldCode="AU" term="%22Zahn+M%22">Zahn M</searchLink>; ProtaGene CGT GmbH, Heidelberg, Germany.<br /><searchLink fieldCode="AU" term="%22Gil-Farina+I%22">Gil-Farina I</searchLink>; ProtaGene CGT GmbH, Heidelberg, Germany.<br /><searchLink fieldCode="AU" term="%22Annoni+A%22">Annoni A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Iaia+S%22">Iaia S</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ott+de+Bruin+LM%22">Ott de Bruin LM</searchLink>; Willem-Alexander Children's Hospital, Department of Pediatrics, Pediatric Stem Cell Transplantation Program, Leiden University Medical Center, Leiden, Netherlands.; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands.<br /><searchLink fieldCode="AU" term="%22Notarangelo+LD%22">Notarangelo LD</searchLink>; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.<br /><searchLink fieldCode="AU" term="%22Pike-Overzet+K%22">Pike-Overzet K</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands.<br /><searchLink fieldCode="AU" term="%22Staal+FJT%22">Staal FJT</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, Netherlands.<br /><searchLink fieldCode="AU" term="%22Villa+A%22">Villa A</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Capo+V%22">Capo V</searchLink>; San Raffaele-Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy.
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  Data: <searchLink fieldCode="JN" term="%22101560960%22">Frontiers in immunology</searchLink> [Front Immunol] 2023 Nov 13; Vol. 14, pp. 1268620. <i>Date of Electronic Publication: </i>2023 Nov 13 (<i>Print Publication: </i>2023).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Frontiers+Research+Foundation]%22">Frontiers Research Foundation] </searchLink><i>Country of Publication: </i>Switzerland <i>NLM ID: </i>101560960 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>1664-3224 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2216643224%22">16643224 </searchLink><i>NLM ISO Abbreviation: </i>Front Immunol <i>Subsets: </i>MEDLINE
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