Article
Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector.
Blood - 15 Sept 2003
Scallan Ciaran D, Lillicrap David, Jiang Haiyan, Qian Xiaobing, Patarroyo-White Susannah L, Parker Amy E, Liu Tongyao, Vargas Joseph, Nagy Dea, Powell Sharon K, Wright J Fraser, Turner Patricia V, Tinlin Shawn J, Webster Sandra E, McClelland Alan, Couto Linda B
Abstract excerpt
Gene therapy for hemophilia A requires efficient delivery of the factor VIII gene and sustained protein expression at circulating levels of at least 1% to 2% of normal. Adeno-associated viral type 2 (AAV2) vectors have a number of advantages over other viral vectors, including an excellent safety profile and persistent gene expression. However, a major disadvantage is their small packaging capacity, which has...
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