Skeletal myoblast-mediated gene transfer for hemophilia B gene therapy

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