Article
Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Sept 2001
Chao H, Monahan P E, Liu Y, Samulski R J, Walsh C E
Abstract excerpt
We previously reported that direct intramuscular injection of non-serotype-2 AAV vectors, especially AAV serotype 1 (AAV1), resulted in expression of supranormal levels of canine F9 in immunodeficient mice. Here we test the ability of the AAV1-F9 vector to deliver sustained expression and correction of factor IX (FIX) deficiency in genetically engineered hemophilic mice. Intramuscular injection of AAV1-F9...
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