Abstract
Recombinant adeno-associated viral (AAV) vector is a promising gene-based strategy for the treatment of several inherited diseases. Using AAV serotype 2 (AAV-2), the most common tested vector in humans, we have determined that the risk of germline transmission and the immune responses to both transgene product and/or vector-capsid proteins are critical obstacles to the safety of this strategy (
Nat Med
12
:342
, 2006
Author notes
Disclosure: No relevant conflicts of interest to declare.
2007, The American Society of Hematology
2007
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