| 摘要 |
Two mechanisms are provided for improving the expression of Factor IX in gene therapy vectors. The first is the use of a specific Factor IX polynucleotide coding sequence designed for optimal expression. The second is the use of transcriptional regulatory regions minimized in size so that they can be used to express Factor IX, as well as any other gene of interest, in a size-constrained environment such as in a self complementary gene therapy vector system.
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