{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["9(22)"],"submitter":["Meng X"],"pubmed_abstract":["<h4>Abstract</h4>Recombination activating gene 1 (RAG1) is essential for variable diversity joining recombination during early T- and B-cell development. Null mutations cause a complete block in receptor rearrangement, resulting in T-B- severe combined immunodeficiency (SCID). Patients with RAG1-SCID require hematopoietic stem cell transplantation for survival. Our phase I/II clinical trial (NCT04797260) is currently evaluating lentiviral RAG1 gene addition in autologous hematopoietic stem and progenitor cells (HSPCs). However, studying early human T-cell development is challenging due to limited access to thymic tissue. The artificial thymic organoid (ATO) system offers a promising in vitro model to study human T-cell differentiation. Here, we show that ATO cultures efficiently support T-"],"journal":["Blood advances"],"pagination":["5920-5934"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12666348"],"repository":["biostudies-literature"],"pubmed_title":["RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids."],"pmcid":["PMC12666348"],"pubmed_authors":["van der Holst R","Berghuis D","Lankester AC","Staal FJT","Meng X","van Eggermond M","Langerak AW","de Kivit S","Vloemans S","Cante-Barrett K","Pike-Overzet K","de Mooij B","Ott de Bruin LM","Melsen JE"],"additional_accession":[]},"is_claimable":false,"name":"RAG1 lentiviral gene therapy restores T-cell development of RAG1-SCID patient cells in artificial thymic organoids.","description":"<h4>Abstract</h4>Recombination activating gene 1 (RAG1) is essential for variable diversity joining recombination during early T- and B-cell development. Null mutations cause a complete block in receptor rearrangement, resulting in T-B- severe combined immunodeficiency (SCID). Patients with RAG1-SCID require hematopoietic stem cell transplantation for survival. Our phase I/II clinical trial (NCT04797260) is currently evaluating lentiviral RAG1 gene addition in autologous hematopoietic stem and progenitor cells (HSPCs). However, studying early human T-cell development is challenging due to limited access to thymic tissue. The artificial thymic organoid (ATO) system offers a promising in vitro model to study human T-cell differentiation. Here, we show that ATO cultures efficiently support T-","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Nov","modification":"2026-06-05T21:50:23.473Z","creation":"2026-05-22T03:15:20.706Z"},"accession":"S-EPMC12666348","cross_references":{"pubmed":["40829114"],"doi":["10.1182/bloodadvances.2025016970"]}}