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血红蛋白疾病是由于血红蛋白分子突变造成其结构或合成异常引起的一类疾病,分为血红蛋白病和地中海贫血两大类。前者表现为血红蛋白分子的珠蛋白肽链结构异常,如镰刀状贫血;后者表现为珠蛋白肽链合成速率的降低,如β-地中海贫血。本文主要以β-地中海贫血和镰刀状贫血为例,从DNA水平、RNA水平和基因调控及干细胞移植等方面介绍血红蛋白疾病基因治疗的研究进展,并结合生命科学的最新发现,对该领域将来可能出现的新的治疗方法提出展望。
Hemoglobin disease is due to the mutation of hemoglobin caused by its structural or synthetic abnormalities caused by a class of diseases, divided into hemoglobin disease and thalassemia major categories. The former is characterized by structural abnormalities in the globin chain of hemoglobin, such as sickle-shaped anemia; the latter is manifested as a decrease in the rate of globin peptide synthesis, such as β-thalassemia. In this paper, β-thalassemia and sickle-like anemia are taken as examples to introduce the research progress of gene therapy of hemoglobin diseases from DNA level, RNA level and gene regulation and stem cell transplantation. Combined with the latest findings of life science, The emergence of new treatments put forward the prospect.