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The target organ for AAV gene therapy influences the risk of immune responses, such as antibody formation to therapeutic proteins.
To the editor: The target organ substantially influences the risk of immune responses to therapeutic proteins in gene therapy. For example, intramuscular administration of an adeno-associated viral (AAV) vector causes robust antibody formation to human coagulation factor IX (hF.
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Cao et al. (2008) studied this question.
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