Skeletal myoblast-mediated gene transfer for hemophilia B gene therapy  

作　　者：K.Kurachi 

机构地区：[1]Department of Human Genetics,University of Michigan Medical School,Ann Arbor,MI,USA

出　　处：《中国实验血液学杂志》1997年第3期328-328,共1页Journal of Experimental Hematology

摘　　要：Myoblast-mediated gene transfer approach has agreat potential to be developed into a durable genetherapy method for various diseases. To increase theexpression level of human factor Ⅸ (hFⅨ) in musclecells, we constructed a series of hFⅨ expression vectorswith the retroviral vector frame and examined their hFⅨexpression in skeletal muscle cells. Firstly, we introducedhFⅨ minigene (hFⅨ ml or hFⅨ m2), which contains atruncated first intron (1. 4 kb or 0. 3 kb) of hFⅨ

关 键 词：SKELETAL INTRON promoter maintained battery INJECTIONS persistent SCID implanted stably 

分 类 号：R456[医药卫生—治疗学]