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Thousands of U-S men have hemophilia (hee-moe-feel-ee-uh), a disorder in which blood doesn’t clot properly. In fact, a clotting factor transfusion is required before these patients can have a tooth pulled or undergo surgery, because they are so prone to profuse bleeding that even a
simple cut can be life-threatening.
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Now University of Florida researchers have found a way to correct the condition in lab mice, stopping excessive bleeding by introducing a segment of D-N-A that encodes for the clotting protein they’re missing. To do this, scientists used a transposon (trans-poze-onn),
a sliver of D-N-A derived from fish that can efficiently transport new genetic information into the genome. Currently, the only safe treatment for hemophilia is a purified form of the protein, but it can cost patients thousands of dollars and its effects don’t last long. Scientists have been trying to find a safe way to perform gene therapy in these patients for years, but problems with the viruses typically used to transport genes have stymied their success. Researchers think the transposon method may represent another avenue for gene therapy.
Dr. Brad Fletcher / UF blood disorders researcher
“This approach of transposon-based gene delivery could be used with a wide range of different physical techniques to get the DNA into the cell. Once the DNA is into the cell, this approach could be used to then integrate it into the genome so it’s long term expressed.”
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The clotting D-N-A was introduced into cells that line the blood vessels. But doctors think corrective genes could someday be introduced into other organs as well, such as the kidney, the heart or even the brain. Researchers also say the method could be evaluated for the treatment of other medical problems, such as graft rejection in organ transplant recipients.
Dr. Brad Fletcher / UF blood disorders researcher
“We have some interesting data on the use of this to prevent graft rejection. Let’s say doctors want to transplant a kidney from a cadaver or donor, you could use this technique to deliver a gene to that organ that would prevent rejection by the immune system of the recipient.”
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At the University of Florida Health Science Center, I’m Mike Garrison
