地中海贫血的基因治疗新进展  

Recent advance in gene therapy for thalassemia

作　　者：蒋满波[1] 曾敏慧[1] 张滨[1] 蔡柳洪[1] 

机构地区：[1]中山大学附属三院不育与性医学科,广州510630

出　　处：《国际遗传学杂志》2014年第2期77-81,共5页International Journal of Genetics

基　　金：国家自然科学基金（81170533）

摘　　要：地中海贫血（简称地贫）是最常见的血液系统遗传病之一，在我国主要分布于长江以南各省，其中两广、云贵及海南为高发省区。重型地贫不仅对患者的生活质量造成巨大的影响，而且为社会和家庭带来巨大的经济负担。输血、除铁和骨髓移植等是地贫的传统治疗方法，近年来由于干细胞技术的发展，地贫的基因治疗有了长足的进步。本文就地贫的基因治疗进行综述。Thalassemia is one of the most common genetic blood diseases, mainly distributing in South China. Thalassemia major not only causes huge negative impact on the patient ＇ s quality of life, but also brings huge economic burden for society and family. Blood transfusion, iron chelation therapy and bone marrow transplantation are traditional treatment methods for thalassemia. In recent years, basing on the development of stem cell technology , gene therapy for tbalassemia has developed very fast. Here we review the development of gene therapy for thalassemia.

关 键 词：地中海贫血 基因治疗 诱导多功能干细胞 

分 类 号：R556.61[医药卫生—血液循环系统疾病]