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Revolutionising healing: Gene Editing's breakthrough against sickle cell disease

7 Mar 2024

Abstract excerpt

Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel...

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PMID: 38493007
DOI: 10.1016/j.blre.2024.101185

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