Gene Therapy for Hereditary Hematological Disorders: From Clinical Breakthroughs to Future Horizons
27 August 2026 at 08:00
Gene therapy is transforming hereditary hematological disorders. This review summarizes approved gene addition, editing, and silencing strategies for sickle cell disease, thalassemia, and hemophilia, highlights curative potential, and discusses remaining challenges such as immune responses, cost, and accessibility