A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward into development. Evaluation of the different combinations in mice that lack factor VIII demonstrated the significant and differing effects the vector components had on liver-specific expression of the hFVIII transgene, as reported in Human Gene Therapy.
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