Audacity of gene therapy

Abstract

Since the identification of DNA as the hereditary material, therapeutic advancements have focused on gene therapy (GT) and allogeneic hematopoietic cell transplants for inherited diseases. Despite successes in treating conditions like β-thalassemia 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 GT to fully realize its potential.