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- Sickle cell disease affects 20 million people globally, with current treatments showing limitations and only 15% of eligible patients having access to matched sibling donors for bone marrow transplants. - Gene therapy emerges as a promising alternative with 85-90% of patients experiencing freedom from severe vaso-occlusive crises, though accessibility and manufacturing time remain significant challenges. - Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) shows potential as an alternative treatment option, particularly for patients without matched donors, with ongoing research to improve outcomes.
