β-地中海贫血的基因治疗研究

地中海贫血是最常见的单基因遗传病,它是由于珠蛋白肽链合成量之间不平衡导致,所以纠正珠蛋白肽链失衡成为地中海贫血治疗研究的关键。临床常采用输血并配合除铁剂,脾摘除,造血干/祖细胞移植等方法治疗地中海贫血,尽管同种异基因移植和姑息治疗已经取得了显著的进步,由于很多因素的限制,大部分患者依然受到死亡的威胁。本文对地中海贫血基因治疗的研究进展做一综述。

The Development in β-thalassemia for Gene Therapy

The hemoglobin disorders of thalassemia is the most prevalent group of human monogenic diseases,which results from globin protein chain peptide synthesis imbalance.Therefore,it becomes the key in treatment of thalassemia to correct this imbalances.In clinical routine treatment,thalassemia is often used with blood transfusion and iron chelation therapy,spleen removed,hematopoietic stem/progenitor cell transplantation methods.Although curative allogeneic stem cell transplantation therapy and palliative therap...