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Gene therapy advance treats hemophilia in mouse models

utographa californica and put it on to a modified type of lentivirus called feline immunodeficiency virus (FIV). FIV causes leukemia in cats but no disease in humans.

The hybrid vehicle efficiently bound to receptors on the liver cells because the modified baculovirus coat serves as a "key" that fits into the "lock," or receptor. The percentage of liver cells that took up the virus increased from approximately 5 percent to 20 percent.

The team also modified the part of the FIV that expresses the therapeutic gene so that its promoter that causes gene expression worked only when it was in a liver cell.

"Even if this FIV modified virus goes to other organs of the body, it won't express well because its promoter is liver-specific," McCray said. "This modification helps prevent negative side effects. For example, if the gene were expressed in immune cells instead of liver cells, it could lead to a damaging immune response."

McCray said the team now is studying additional ways to make the hybrid vector express the protein even better.


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Source:University of Iowa


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