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Efficient Production of Dual Recombinant Adeno-Associated Viral Vectors for Factor VIII Delivery

1 Aug 2014

Abstract excerpt

Recombinant adeno-associated viral (rAAV) vectors have gained attention for human gene therapy because of their high safety and clinical efficacy profile. For factor VIII gene delivery, splitting the coding region between two AAV vectors remains a viable strategy to avoid the packaging capacity limitation (∼5.0 kb). However, it is time-consuming and labor-intensive to produce two rAAV vectors in separate batches....

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PMID: 25093498
DOI: 10.1089/hgtb.2014.093

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