Cure for Hemophilia

Cure for HemophiliaThe cure for hemophilia is my favorite thing to talk about! Isn't that what we are all waiting, hoping and praying for?

Most of the research for the cure for hemophilia has been centered around “Gene Therapy.” Hemophilia gene therapy is simply amazing. I have heard many doctors speak with a lot of hope for the future. To explain gene therapy I need to first explain a little about hemophilia.

People with hemophilia have low levels of certain blood clotting proteins, usually factor 8 or factor 9 (factor VIII or factor IX), which are necessary for clotting of the blood. These blood clotting proteins are produced continually in a person without hemophilia.

Most of these proteins are produced in our livers. But other cells contribute to the production also. Hemophilia is on the top of the list of disorders that may by cured with gene therapy because of it’s simplicity. It is only one missing blood clotting protein caused by one genetic mutation.

Basically, hemophilia gene therapy should work like this. Scientist remove cells from a person with hemophilia. Usually liver cells, but other cells, muscle, fat etc. have proven to be successful. The scientists then alter the cells genetically by inserting new genetic material into them that reprograms the cells.

This new genetic material instructs the cells to produce factor 8 or 9 (factor VIII or factor IX). The cells are then reinserted into the person with hemophilia and viola! The person with hemophilia no longer has hemophilia.

Sounds simple, and there have been many successes with both animals & humans. There is an entire colony of dogs with hemophilia in Chapel Hill, North Carolina. There have also been many experiments with mice. There have also been several experiments with humans.

Hemophilia gene therapy works. The genetically altered cells produce factor. The cells survive and reproduce. Some of the 1990’s experiments maintained factor levels for several months. Now the results are even more promising.

Some of the trials have maintained blood clotting factor levels for over 3 years and these trials are ongoing. These animals are still being studied. It is only a matter of time before these processes are perfected. A day will come when a person with hemophilia will go to a treatment center and leave without hemophilia.
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