Advances in Gene Therapy for Rare Immune Disorders

Gene therapy has moved from experimental trials to approved treatments for several rare immune disorders. By introducing a functional copy of a defective gene into a patient's own cells, we can potentially correct the root cause of disease rather than only managing symptoms. Recent approvals and late-stage trials focus on conditions such as severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and certain forms of chronic granulomatous disease. Lentiviral and adeno-associated virus (AAV) vectors have improved safety and efficacy profiles, though long-term follow-up remains critical. Challenges include cost, access, and ensuring equitable delivery across healthcare systems. This article reviews the latest clinical data and what patients and families can expect from the next generation of gene-based therapies.