Hyper-Active Factor Ix Vectors For Liver-Directed Gene Therapy Of Hemophilia 'B' And Methods And Use Thereof


Ontology type: sgo:Patent     


Patent Info

DATE

N/A

AUTHORS

VANDENDRIESSCHE, THIERRY , CHUAH, MARINEE

ABSTRACT

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. More... »

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