HYPER-ACTIVE FACTOR IX VECTORS FOR LIVER-DIRECTED GENE THERAPY OF HEMOPHILIA 'B' AND METHODS AND USE THEREOF

摘要

The present invention relates to vectors containing liver-specific regulatory sequences and a factor IX gene containing a hyper-activating mutation, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and hyper-active factor IX genes are also disclosed. The present invention is particularly useful for applications using gene therapy.