Audacity of Gene Therapy

Since the identification of DNA as the hereditary material, therapeutic advances have focused on gene therapy and allogeneic haematopoietic cell transplants for inherited diseases. Despite successes in treating conditions like β-thalassaemia and sickle cell disease, challenges such as insertional oncogenesis and immunogenicity persist. Future efforts must prioritize improving vector safety, refining manufacturing processes and expanding access to gene therapy, to fully realize its potential.