β-地中海贫血的新见解

E. Paubelle, X. Thomas
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引用次数: 0

摘要

地中海贫血是由血红蛋白产生缺陷引起的低色素小细胞贫血的遗传原因。β-地中海贫血是由β-珠蛋白链产生减少引起的,可影响多个器官,并与相当高的发病率和死亡率相关。这篇综述旨在强调在无效红细胞生成控制、金属螯合和基因治疗方面取得的重大进展,这将带来新的希望,并将在不久的将来改变患者的管理和预后。
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New Insights in β-Thalassaemia
Thalassaemia is a hereditary cause of hypochromic microcytic anaemia resulting from defects in haemoglobin production. β-thalassaemia, which is caused by a decrease in the production of β-globin chains, affects multiple organs and is associated with considerable morbidity and mortality. This review aims to highlight the significant progress being made in the areas of ineffective erythropoiesis control, metal chelation, and gene therapy, which is bringing new hope and should change patient management and prognosis in the near future.
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