Liver specific transcriptional enhancer

Details Liver specific transcriptional enhancer Liver specific transcriptional enhancer

2007-02-20

2007-02-20

Priebe, Scott D. (Department: 1633)

Organic compounds -- part of the class 532-570 series

Organic compounds

Carbohydrates or derivatives

C435S320100, C536S023100

Reexamination Certificate

active

10885457

ABSTRACT:
Recombinant lentiviruses and transfer vectors for transgene delivery as well as methods for gene therapy using such vectors are disclosed. The invention provides a third generation lentiviral packaging system and a set of vectors for producing recombinant lentiviruses, as well as novel tissue specific enhancer and promoter elements useful for optimizing liver specific transgene delivery. The transgene is preferably a blood clotting factor such as human factor IX (hFIX) or human factor VIII (hFVIII) and can be used for treatment of hemophilia.

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