Article
A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype.
Journal of thrombosis and haemostasis : JTH - 1 Feb 2003
Sarkar R, Xiao W, Kazazian H H
Abstract excerpt
A major obstacle for delivery of factor (F)VIII using adeno-associated virus (AAV) vectors is the large size of FVIII cDNA, which is well above the 5 kb packaging limit for AAV. Here we construct a < 5 kb FVIII-AAV vector using murine FVIII cDNA and a strong liver-specific albumin promoter. We assessed the efficacy of this vector using three different routes of administration, intraportal, intrasplenic and tail...
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