Hemophilia Gene Therapy

HIV-1 Based Vectors Result in Therapeutic Levels of Factor VIII and IX

Published in Blood Weekly, September 28th, 2000

According to a study conducted in the United States, HIV-1 based vectors successfully produced therapeutic levels of human factor VIII and IX in a murine model.

"Lentiviral vectors have the potential to play an important role in hemophilia gene therapy," stated F. Park and colleagues, Stanford University, California. "The present study used human immunodeficiency virus (HIV) based lentiviral vectors containing an EF1 alpha enhancer/promoter driving human factors VIII (hFVIII) or IX (hFIX) complementary DNA expression for portal vein injection into C57B1/6 mice."

They reported that increased doses of hFIX-expressing lentivirus resulted in a...

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Source: Blood Weekly (2000-09-28)

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