遗传校正β-地中海贫血患者特异性iPSCs的功能探讨  

Investigate the function of corrected β-thalassemia patient-specific iPSCs in transplanted SCID mice

作　　者：周林[1] 郑陈光[1] 杨泽[2] 

机构地区：[1]广西壮族自治区妇幼保健院,530003 [2]卫生部北京医院,卫生部北京老年医学研究所,100730

出　　处：《中国老年保健医学》2012年第6期27-28,共2页Chinese Journal of Geriatric Care

基　　金：广西自然科学基金项目(编号:桂科自0897009)

摘　　要：目的探讨遗传校正后的β-地中海贫血患者特异性iPSCs在SCID移植小鼠中的功能。方法将遗传校正的iPS细胞移植入SCID小鼠,移植6周后,收集外周血、移植与非移植的骨髓细胞及脾血细胞,使用特异性人类HLAABC抗体和人类CD71抗体进行流式细胞计数分析,检测SCID的移植细胞的红细胞生成能力;采用westernblot技术分析其β-珠蛋白和γ-珠蛋白的表达情况。结果移植入SCID小鼠体内的iPS细胞具有生成红细胞能力,可以提高SCID小鼠的红细胞生成量,还可以产生人类特异性β-珠蛋白与γ-珠蛋白。结论校正后的iPSCs具有潜在的临床治疗重症β-地中海贫血的功能。Objective To investigate the function of the genetic corrected β-thalassemia patient-specific iPSCs in transplanted SCID mice.Methods Transplanted the genetically corrected iPSCs into SCID mice.Six weeks after transplantation,peripheral blood,bone marrow （BM） cells from the transplantation and the nontransplantation sides or spleen cells were collected and analyzed to detect the ability of differentiation of iPSCs by flow cytometry using specific human HLA ABC antibody and human CD71 antibody,separately.And then western blot was used to analyse the human β-globin and γ-globin expression of of iPSCs.Results The corrected iPSCs have the ability of differentiation and could improved the red blood cell production.Moreover,the generation of human β-globin and γ-globin could be detected in vivo of the SCID mice transplanted with corrected β-thalassemia patient-specific iPSCs.Conclusion Our study provides strong evidence that the genetic corrected iPSCs have potential clinical uses in treating β-thalassemia major.

关 键 词：Β-地中海贫血 IPSCS 移植 功能 探讨 

分 类 号：R556.61[医药卫生—血液循环系统疾病]