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Leukocyte adhesion deficiency type 1 (LAD1) is a primary immunodeficiency that occurs due to inherited mutations in ITGB2, which encodes the β2 common integrin subunit CD18, and results in the inability of leukocytes to migrate from the bloodstream to sites of infection. Patients exhibit frequent life-threatening infections and a high mortality risk, and the only curative treatment is allogeneic hematopoietic stem cell transplantation (HSCT). Booth et al (N Engl J Med 2025;392:1698-1709; https://doi.org/10.1056/NEJMoa2407376) conducted a phase 1-2 single-group study examining lentiviral-based gene therapy in LAD1.