Construction Of RAAV2-GPIIb/GpIIIa Vector And Test Of Its Expression And Function In Vitro

Glanzmann thrombasthenia is a rarely congential hereditary autosomal recessive hemorrhagic disorder, characterized by a severe reduction or absence of platelet aggregation in response to multiple physiologicagonists, such as ADP thrombin collagen and adrenalin, due to qualitation or quantitative abnormalities of platelet glycoprotein GP â…¡b/â…¢a complex, which are caused by gene defects in megakarocyte. Platelet can not form platelet embolism in physiologic hemostasia â…¡ phase.The main clinical manifestations are bleeding symptoms in different degree from child, nosebleed skinpurpuric, even including fatal excessive bleeding. No regularly routine therapy is highly recommended, and repeatly transfusion would make patients refractory to platelets.Gene therapy is one potential way in the treatment of Glanzmann thrombasthenia by using genetic therapeutic vector to transinfect hematopoietic stem cell to correct the defective gene in vitro and transfusing the target cells to the affected individual.Choosing a kind of safty and high efficient gene transfer system is the key of successful gene therapy. Wilcox et al began this study by using retrovirus vector in 1999, but safety problem of vector retrovirus restrict its use, so we should find another vector. Recombinant adeno-associated virus (rAAV) vector is one of the most ideal vectors for gene therapy due...