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Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
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- Science Translational Medicine, 2024, v. 16, n. 733, p. 1, doi. 10.1126/scitranslmed.adh8162
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Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations.
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- Frontiers in Immunology, 2023, p. 1, doi. 10.3389/fimmu.2023.1268620
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- Article