April 5, 2016 – Genes and Hemophilia

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Anchor lead: Gene therapy may one day soon cure hemophilia, Elizabeth Tracey reports

Hemophilia can usually be managed well with infusions of missing blood clotting factors, most often factor XIII and factor IX. Yet the schedule is onerous, requiring infusions into a vein every other day or every few days.  Clifford Takemoto, a hemophilia expert at Johns Hopkins, says gene therapy offers the best hope for cure for those with the bleeding disorder.

Takemoto: This is a disorder where it’s a problem with a single gene. So theoretically you should be able to fix it with gene therapy and there actually are a number of trials using different strategies to use gene therapy to correct the defect in factor IX deficiency.  It turns out the gene for factor IX is much smaller so technically it’s much less challenging to be able to do the gene therapies.  Some of them look promising but they’re not ready for the general public just yet.  :26

Takemoto says even when gene therapy is up and running, those who’ve been managing well with infusions may choose to simply continue that treatment course.  At Johns Hopkins, I’m Elizabeth Tracey.