因镰状细胞疾病将β球蛋白基因转移至人骨髓
Introduction
Sickle cell disease (SCD) is one of the most common monogenic disorders worldwide and is a major cause of morbidity and early mortality (1). Although SCD is well characterized, there is still no ideal long-term treatment. Current therapies are based on induction of fetal hemoglobin (HbF) to inhibit polymerization of sickle hemoglobin (HbS) (2) and cell dehydration (3) or reduction of the percentage of HbS by transfusions (4). Allogeneic HSC transplantation (HSCT) from BM or umbilical cord blood (UCB) is a potentially curative therapy, although only a small percentage of patients have undergone this procedure, mostly children with severe symptoms who had HLA-matched siblin
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来源: J Clin Invest.
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