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Gene therapy for sickle cell disease and thalassemia

27 Feb 2025

Abstract excerpt

PURPOSE OF REVIEW: Thalassemia and sickle cell disease are among the most frequent monogenic hereditary diseases. Access to transfusions, iron chelation therapies and drugs such as hydroxyurea have improved life expectancy and quality of life. However, these diseases still cause significant disability. The first available curative therapy, bone marrow transplantation, is unfortunately not feasible for all...

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PMID: 40013946
DOI: 10.1097/moh.0000000000000867

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