This has recently been reported for patients with Hemophilia B.  Hemophilia B, also known as Christmas disease, is due to a deficiency of the clotting factor IX (FIX).  The first reported case of Hemophilia B due to a decrease in FIX was in 1952, and was called “Christmas Disease” after the first patient diagnosed was named Stephen Christmas.  Without this clotting factor, the blood does not form clots and results in severe bleeding episodes, especially in the joints and muscles.

Bettert reatment for this disorder began back in the 1960’s where they would inject FIX concentrates into the blood of patients with hemophilia B.  This increased the average age of death of 24 to a median lifespan of 63 years of age.  So with the success of the protein therapy, why try to fix the genes?  With each treatment costing $150,000 to $300,000, a patient needing clotting factors for hemophilia could incur a lifetime cost of $20 million.

So there needs to be a way that a patient can have a more effective treatment option that will cost less.  This new treatment option offers some hope.  Using a new virus for the administration of the gene, patients have seen an increased production of FIX protein for longer periods of time, and were able to stop or decrease the amount of concentrate injections they would need.  With one injection of the virus only costing about $30,000, dramatic cost savings have already been seen.  While this does offer new hope for the treatment of clotting disorders, follow-up with a larger number of patients and for longer periods of time will be needed to fully weigh the benefits and risks of this technique.  Once this has been done, hopefully we will see gene therapy used more in practice and maybe even for more than just clotting disorders.

Ponder, Katherine P.  Merry Christmas for Patients with Hemophilia B. The New England Journal of Medicine 10.1056; December 10, 2011.  Nathwani A.C., Tuddenham E.G.D., Rangarajan S., et al.

Interestingly enough, the Spruce, a very common Christmas tree species, has seven times more DNA than a human. How is that, you say?  They are but simple trees, and we are complex animals with skills and intellect beyond compare! Well, you may not say that, but my students do! I shared that little gem with my class today, and they were shocked.

It turns out that all conifers like the Spruce, have 12 chromosomes (humans have 46), but they are really big. Scientists aren’t sure why this is so, but some speculate that this large amount of DNA may be how they have been such a successful plant, surviving for millions of years. There are several plant species that have way more DNA than we do, which brings to light the questions: Does more DNA make an organism more complex? Could a plant be more complex than a human? I suppose that depends on who you ask.

What I do know is that I love my Christmas tree, and all 12 of its giant chromosomes!

To read more about the Spruce geneome project in Sweden, go to: http://www.innovations-report.com/html/reports/agricultural_sciences/christmas_tree_times_dna_time_map_145376.html