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Article

Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B

25 Jan 2014

Abstract excerpt

BACKGROUND: Self-complementary adeno-associated virus (scAAV) vectors have become a desirable vector for therapeutic gene transfer due to their ability to produce greater levels of transgene than single-stranded AAV (ssAAV). However, recent reports have suggested that scAAV vectors are more immunogenic than ssAAV. In this study, we investigated the effects of a self-complementary genome during gene therapy with a...

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PMID: 24460861
DOI: 10.1186/1479-5876-12-25

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