基于造血干细胞为靶细胞的基因治疗  

Research progress of hematopoietic stem cells as target of gene therapy

作　　者：张铸业[1] 于慧慧[1] 王彦刈[1] 

机构地区：[1]贵州大学生命科学学院,贵阳550025

出　　处：《生命的化学》2011年第1期41-48,共8页Chemistry of Life

摘　　要：在基因治疗中,造血干细胞因为具有自我更新及分化为各种血细胞系的能力而成为一种很有吸引力的靶细胞。将外源目的基因导入造血干细胞,以纠正或补偿因基因缺陷和异常引起的疾病,特别是血液疾病已取得重要进展,例如:腺苷脱氨酶缺陷病、血友病、地中海贫血症及镰状细胞性贫血症等。而慢病毒以其转染效率高,能够感染非分裂期细胞的特点成为转染造血干细胞的最适合载体,本文就造血干细胞的特性、载体的选择及临床应用和基因治疗的安全性等方面作一综述。In gene therapy, hematopoietic stem cells are arguably the most attractive target cell population because of their ability to replenish all blood cell types （multipotency） and their ability to self-renew. The exogenous gene will be transferred into hematopoietic stem cells for treating diseases by correcting the defects of genes. Important research progress has been made in blood diseases, such as ADA-deficient SCID, hemophilia, thalassemia and sicklemia. The lentiviral vectors have been the most suitable vectors because they can transfect quiescent hematopoietic stem cells more effectively than any other vectors. This paper summarized the characteristics of hematopoietic stem cells, the choice of vectors, the clinical application of gene therapy and the safety of gene therapy.

关 键 词：造血干细胞 基因治疗 慢病毒载体 

分 类 号：R450[医药卫生—治疗学]