Article
Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.
Blood - 1 Dec 2003
Scallan Ciaran D, Liu Tongyao, Parker Amy E, Patarroyo-White Susannah L, Chen Haifeng, Jiang Haiyan, Vargas Joseph, Nagy Dea, Powell Sharon K, Wright J Fraser, Sarkar Rita, Kazazian Haig H, McClelland Alan, Couto Linda B
Abstract excerpt
Using separate adeno-associated viral 2 (AAV2) vectors to deliver the heavy and light chains of factor VIII (FVIII) we have overcome the packaging limitations of AAV, achieving phenotypic correction of hemophilia A in mice. AAV vectors were constructed that use a liver-specific promoter and the cDNA sequences of either the human or canine heavy and light chains of FVIII. After intraportal vein injection of these...
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