摘要
构建了以ＭＣＫ（肌肉肌酸激酶）增强子增强、β－ａｃｔｉｎ启动子启动的、反向插入载体Ｇ１Ｎａ的Ｇ１ＮａＰＡＩＸｉ′ＢＡＭ。在与同一系列的、仅内含子元件有差异的载体Ｇ１ＮａＭＢＡＩＸ和Ｇ１ＮａＭＢＡｉＩＸ的体外体内表达比较证明，反向载体是表达最高最稳定的。进一步的二次体内注射研究表明，此载体是国内已报道的最适于肌细胞途径血友病Ｂ基因治疗的反转录病毒载体。
G1NaPAIXi′BAM, a high expression retroviral vector which has a reverse inserted cassette of MCK enhancer and β actin promoter controled hFIX minigene, has been constructed. Compared with other two related vectors differeent in intron component, this vector shows the highest expression in vitro and in vivo. Further studies, such as second injection,show that this vector is the highest expression retroviral vector of gene therapy for hemophilia B by muscle cells.
出处
《高技术通讯》
EI
CAS
CSCD
1998年第3期38-41,共4页
Chinese High Technology Letters
基金
863计划资助项目
