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Received β€” 10 September 2026 ⏭ Molecular Therapy Nucleic Acids

Gene Therapy for Hereditary Hematological Disorders: From Clinical Breakthroughs to Future Horizons

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
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