中国科学C辑(英文版)
中國科學C輯(英文版)
중국과학C집(영문판)
SCIENCE IN CHINA (Series C)
2003年
4期
422-430
,共9页
陈立%陈浩明%陆华中%吴小兵%卢大儒%邱信芳%薛京伦
陳立%陳浩明%陸華中%吳小兵%盧大儒%邱信芳%薛京倫
진립%진호명%륙화중%오소병%로대유%구신방%설경륜
hemophilia B mouse%gene therapy%AAV%HSV
Recombinant AAV particles of high titer (>1013 virus genome/mL) were prepared according to the rHSV/AAV helper virus method. After intramuscular injection of viral vectors in the hind limb, a sustained elevated level (>370 ng/mL) of murine FIX expression in the plasma of hemophilia B mouse was detected and persisted for more than 350 days. The biological activity reached 30% of normal levels, and bleeding symptoms in the treated mice were significantly alleviated. No anti-FIX antibody (inhibitor) was detected, though anti-AAV antibodies were found at a very low level after single injection. Repeated injection with rAAV/mFIX led to a variation in anti-AAV antibody levels between the two groups which had received different doses. Results from tissue analysis confirmed the skeletal muscle as the origin for circulating functional mFIX. Our results suggest that AAV-mediated gene transfer offers a promising method of gene therapy for hemophilia B.
