Article
Adeno-associated Vector Toxicity—To Be or Not to Be?
1 Nov 2015
Abstract excerpt
Vectors based on adeno-associated virus (AAV) are currently in use in numerous gene therapy clinical trials with reports of benefit for patients, for example in treating hemophilia or inherited blindness. An AAV1 vector (Glybera) has recently been approved as the first gene therapy to achieve marketing authorization in Europe. More than 120 clinical trials using AAV have shown vector safety, with a transient and...
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