Artemis-deficient severe combined immunodeficiency (ART-SCID) patients usually respond poorly to standard bone marrow transplants , leaving gene correction as the only significant alternative for such patients. A study investigated a breakthrough gene therapy in 10 infants with newly diagnosed ART-SCID by infusing autologous CD34+ cells transfected with a lentiviral vector, preceded by targeted low-exposure busulfan. The lentiviral construct design ensured therapeutic levels of Artemis protein and no overexpression.
All 10 patients achieved feasibility criteria at 42 days post-infusion that included corrected peripheral blood count, no clinical bleeding, and no need for platelet transfusion. Four out of the nine patients achieved T-cell immune reconstitution at 12 months and B-cell immunity by 24 months, allowing discontinuation of immunoglobulin replacements. This safe approach…
