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. |
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| 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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