%0 Journal Article
%T Skeletal muscle-specific expression of human blood coagulation factor IX rescues factor IX deficiency mouse by AAV-mediated gene transfer<br>Skeletal muscle-specific expression of human blood coagulation factor Ⅸ rescues factor Ⅸ deficiency mouse by AAV-mediated gene transfer
%A LAI Lihui
%A <br>赖立辉
%A 陈立
%A 卢大儒
%A 王琪
%A 高啸波
%A 邱信芳
%A Jerry L.Hsueh
%A 薛京伦
%A 王健民
%A 周虹
%J 中国科学C辑(英文版)
%D 1999
%I Springer
%X The efficacy of recombinant adeno-associated virus (AAV) vector to deliver and express human blood clotting factor IX (hFIX) gene in skeletal muscle of coagulation factor IX deficiency mouse strain (FactorIX-knockout) is evaluated. The muscle creatine kinase enhancer (MCK) and β-actin promoter (βA) were used to drive the hFIX minigene (hFIXml), which was flanked by AAV inverted terminal repeats (ITRs). Following intramuscular injection of high titer (2.5 × 1011 vector genomes/mL) of rAAV, increased hFIX expression (256 ng/mL of plasma) was achieved. The time course of hFIX expression demonstrated that the expression level gradually increased over a period of two weeks before anti-hFIX antibodies developed in mouse circulating plasma. Those results provided a promising evidence that rAAV-mediated gene transfer and skeletal muscle-specific expression of hFIX is a feasible strategy for treating patients for hemophilia B. Project supported by the State High Technology Development Program (863-Z20-02-01), Shanghai Postdoctoral Foundation and National Natural Science Foundation of China (Grant No. 39880019).
%K AAV
%K hFIX
%K factor IX deficiency mouse
%K direct-muscular gene therapy<br>
%U http://www.alljournals.cn/get_abstract_url.aspx?pcid=90BA3D13E7F3BC869AC96FB3DA594E3FE34FBF7B8BC0E591&jid=180CF3A72E750F3261A8A60EDC957784&aid=B29FE0D9D10F515736EEE0573A6784C5&yid=B914830F5B1D1078&vid=ECE8E54D6034F642&iid=B31275AF3241DB2D&sid=3EABEBD973E45554&eid=20ED669EB429E15C&journal_id=1674-7305&journal_name=ScienceChina.Lifesciences&referenced_num=0&reference_num=15