Jasmeen Dara

Gene therapy approaches for inborn errors of immunity: from bench to bedside

The 30-year evolution of autologous gene therapy as a vital alternative to allogeneic hematopoietic stem cell transplantation for IEIs is explored, tracing the transition from early gamma-retroviral gene addition - which successfully restored immunity in severe combined immunodeficiency but carried high risks of insertional mutagenesis and leukemogenesis - to the adoption of safer self-inactivating lentiviral vectors.