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RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids.

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Title: RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids.
Authors: Meng X; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Melsen JE; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., van der Holst R; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., de Mooij B; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Vloemans S; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., van Eggermond M; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Berghuis D; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., Lankester AC; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands., de Kivit S; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Langerak AW; Department of Immunology, Laboratory Medical Immunology, Erasmus Medical Center, University Medical Center, Rotterdam, The Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands., Ott de Bruin LM; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands., Canté-Barrett K; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands.
Source: Blood advances [Blood Adv] 2025 Nov 25; Vol. 9 (22), pp. 5920-5934.
Publication Type: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Multicenter Study
Journal Info: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids.
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  Data: <searchLink fieldCode="AU" term="%22Meng+X%22">Meng X</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Melsen+JE%22">Melsen JE</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22van+der+Holst+R%22">van der Holst R</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22de+Mooij+B%22">de Mooij B</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Vloemans+S%22">Vloemans S</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22van+Eggermond+M%22">van Eggermond M</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Berghuis+D%22">Berghuis D</searchLink>; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Lankester+AC%22">Lankester AC</searchLink>; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22de+Kivit+S%22">de Kivit S</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Pike-Overzet+K%22">Pike-Overzet K</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Langerak+AW%22">Langerak AW</searchLink>; Department of Immunology, Laboratory Medical Immunology, Erasmus Medical Center, University Medical Center, Rotterdam, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Staal+FJT%22">Staal FJT</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Ott+de+Bruin+LM%22">Ott de Bruin LM</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Stem Cell Transplantation Program and Laboratory for Pediatric Immunology, Willem-Alexander Children's Hospital, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands.<br /><searchLink fieldCode="AU" term="%22Canté-Barrett+K%22">Canté-Barrett K</searchLink>; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Novo Nordisk Foundation Centre for Stem Cell Medicine (reNEW), Leiden University Medical Center, Leiden, The Netherlands.
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  Data: <searchLink fieldCode="JN" term="%22101698425%22">Blood advances</searchLink> [Blood Adv] 2025 Nov 25; Vol. 9 (22), pp. 5920-5934.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22American+Society+of+Hematology%22">American Society of Hematology </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101698425 <i>Publication Model: </i>Print <i>Cited Medium: </i>Internet <i>ISSN: </i>2473-9537 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2224739529%22">24739529 </searchLink><i>NLM ISO Abbreviation: </i>Blood Adv <i>Subsets: </i>MEDLINE
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              Text: 2025 Nov 25
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