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1. Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities

2. Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis

4. Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency

5. Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy

6. T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

8. Unraveling Pathophysiology and Hematopoiesis of Vexas Syndrome By Multi-Omics Analyses and Targeted Gene Editing

9. Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells

11. Lentiviral-Mediated Gene Therapy for the Treatment of Adenosine Deaminase 2 Deficiency

12. In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

13. Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

16. In Vivo Tracking of T Cells in Humans Unveils Decade-Long Survival and Activity of Genetically Modified T Memory Stem Cells

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